Patient-focused drug development meeting reports published
FDA posts condition-specific meeting reports and resources from externally-led PFDD meetings and patient listening sessions.
What this means
The FDA published summaries from patient-focused meetings where people with rare diseases shared their experiences with symptoms, treatments, and daily challenges. These reports cover conditions like Diamond-Blackfan anemia, mitochondrial disorders, and Huntington's disease from discussions held between 2023 and 2026. The insights may help drug developers and regulators better understand patient priorities when reviewing new therapies for these often-overlooked conditions.
From the source
This webpage provides links to certain publicly available external reports and resources relating to patient experience data. The patient community, patient advocates, researchers, drug developers, and federal agencies may find these materials useful. For specific questions related to a report or resource, FDA recommends reaching out to the point of contact listed on the cover page of the report.
This webpage hosts an alphabetical listing of condition-specific meeting reports and other information related to patients’ experience. These meetings include FDA-led Patient-Focused Drug Development (PFDD) meetings, Externally-led PFDD meetings, and Patient Listening Sessions. Other information includes proposed draft guidance relating to patient experience data, natural history studies, or other condition-specific background on condition and discussion of unmet medical need.
FDA’s Office of the Commissioner is responsible for managing the Agency’s Patient Listening Session program. While conditions for which there were Patient Listening Sessions conducted are listed individually on this page, the link will direct you to the Patient Listening Session page where you will be able to see all the Patient Listening Session Summaries posted by the Office of the Commissioner.
For more information regarding what types of resources may be included on this webpage, how to submit a publicly available website link to FDA, and other general questions, please review our Frequently Asked Questions. For EL-PFDD meeting summary reports, we request that links include a cover page or similar opening statement as part of their report or resource to provide information about the authors, funding, and related information.
Please note that although FDA reviews the materials that link to external pages before posting them to ensure that the materials are within the scope of the webpage, FDA does not assess their scientific merit or compliance with regulatory requirements. Our decision to post links to these materials does not reflect an endorsement of their authors, sponsors, or content.
Disease or Condition (alphabetical) Type of Meeting Resource(s) Meeting Date
Diamond-Blackfan Anemia Syndrome (DBAS) EL-PFDD Meeting
Host: The Styrke Foundation for Rare Disease and Treatment
Report pending May 1, 2026
Genetic Cardiomyopathies EL-PFDD Meeting
Host: Hypertrophic Cardiomyopathy Association
Report pending April 23, 2026
Central Nervous System Disseminated Coccidiomycosis (Cocci Meningitis) EL-PFDD Meeting
Host: MYCARE Foundation
Report pending April 14, 2026
Idiopathic Pulmonary Fibrosis (IPF) Patient Listening Session Patient Listening Session Summary April 9, 2026
Infertility and Advancing Therapeutic Solutions for In Vitro Fertilization (IVF) EL-PFDD Meeting Host: Alliance for Fertility Preservation Report pending March 26, 2026
Diabetic Retinal Disease EL-PFDD Meeting Host: Mary Tyler Moore Vision Initiative Report pending February 12, 2026
Mitochondrial Encephalomyopathy Lactic Acidosis and Stroke-Like Episodes Syndrome (MELAS) EL-PFDD Meeting Host: MitoAction Report pending February 10, 2026
Progressive Supranuclear Palsy (PSP) EL-PFDD Meeting Host: Cure PSP Report pending February 6, 2026
Wilson Disease (WD) EL-PFDD Meeting Host: Wilson Disease Association Report pending January 29, 2026
CACNA1A Patient Listening Session Patient Listening Session Summary January 22, 2026
Becker Muscular Dystrophy Patient Listening Session Patient Listening Session Summary January 14, 2026
Familial Alzheimer’s Disease (FAD) Patient Listening Session Patient Listening Session Summary December 10, 2025
Alexander Disease EL-PFDD Meeting Host: End Alexander Disease Report pending December 5, 2025
Glycogen Storage Disease (GSD1a) EL-PFDD Meeting Host: The Children’s Fund for GSD Research Report pending October 9, 2025
Congenital Erythropoietic Porphyria (CEP) EL-PFDD Meeting Host: United Porphyrias Association Report pending October 14, 2025
Multiple Sclerosis (MS) EL-PFDD Meeting Host: National Multiple Sclerosis Society Report pending October 29, 2025
Juvenile onset forms of GM1 gangliosidosis and GM2 gangliosidoses (Tay-Sachs and Sandhoff) Patient Listening Session Patient Listening Session Summary August 28, 2025
Chronic Granulomatous Disease (CGD) Patient Listening Session Patient Listening Session Summary August 11, 2025
APOE e4 Alzheimer’s Disease Patient Listening Session Patient Listening Session Summary July 22, 2025
Leukoencephalopathy with Calcifications and Cysts (LCC) Patient Listening Session Patient Listening Session Summary July 11, 2025
MAPT Frontotemporal Dementia (MAPT-FTD) Patient Listening Session Patient Listening Session Summary May 23, 2025
Wolfram Syndrome Patient Listening Session Patient Listening Session Summary May 21, 2025
MECP2 Duplication Syndrome (MDS) Patient Listening Session Patient Listening Session Summary April 3, 2025
Leukoencephalopathy and Brain Stem Involvement and Lactate Elevation (LBSL) EL-PFDD Meeting Host: Cure LBSL Report pending August 1, 2025
Adult Dysphagia EL-PFDD Meeting Host: National Foundation of Swallowing Disorders Meeting Report June 10, 2025
Adult-Onset Leukoencephalopathy with Axonal Spheroids and Pigmented Glia (ALSP) EL-PFDD Meeting Host: Sisters’ Hope Foundation Report pending June 20,2025
Myhre Syndrome Patient Listening Session Patient Listening Session Summary December 3, 2024
Spinal Muscular Atrophy (SMA) N/A SMA Community Risk Tolerance Update: Comparison of 2022 and 2017 SMA Risk/Benefit Survey Data N/A
Shwachman-Diamond Syndrome (SDS) EL-PFDD Host: Shwachman-Diamond Syndrome Alliance Meeting Report June 4, 2025
Developmental and Epileptic Encephalopathy (DEE) Patient Listening Session Patient Listening Session Summary November 22, 2024
DLG4-related synaptopathy Patient Listening Session Patient Listening Session Summary July 17, 2024
Facioscapulohumeral Muscular Dystrophy (FSHD) Patient Listening Session Patient Listening Session Summary August 23, 2024
MEF2C Haploinsufficiency Syndrome (MCHS) Patient Listening Session Patient Listening Session Summary September 19, 2024
Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) Patient Listening Session Patient Listening Session Summary June 5, 2024
Okur-Chung Neurodevelopmental Syndrome (OCNDS) Patient Listening Session Patient Listening Session Summary July 23, 2024
Phenylketonuria (PKU) EL-PFDD Meeting Host: National PKU Alliance (NPKUA) Meeting Report May 8, 2025
Non-ketotic Hyperglycinemia (NKH) Patient Listening Session Patient Listening Session Summary December 15, 2023
Adult-Onset Leukoencephalopathy with Axonal Spheroids and Pigmented Glia (ALSP) Patient Listening Session Patient Listening Session Summary January 17, 2024
Sarcoidosis EL-PFDD Meeting Host: Foundation for Sarcoidosis Research Meeting Report October 28, 2024
Pre-symptomatic and Early to Mid-stage Adult Onset of Huntington’s Disease (HD) EL-PFDD Meeting Host: Huntington’s Disease Society of America (HDSA) Meeting Report November 13, 2024
Dementia with Lewy Bodies (DLB) EL-PFDD Meeting Host: Lewy Body Dementia Association Report pending October 15, 2024
Lupus Nephritis (LN) EL-PFDD Meeting Host: National Kidney Foundation Report pending October 11, 2024
Classic Congenital Adrenal Hyperplasia (CAH) EL-PFDD Meeting Host: CARES Foundation Meeting Report October 1, 2024
Thalassemia EL-PFDD Meeting Host: Cooley’s Anemia Foundation Report Unavailable July 7, 2017
Neurofibromatosis Type 1 (NF1)/ Cutaneous Neurofibroma (cNF) Patient Listening Session Patient Listening Session Summary May 21, 2024
KCNT1-Related Epilepsy Patient Listening Session Patient Listening Session Summary April 22, 2024
Attention-Deficit/Hyperactivity Disorder (ADHD) Patient Listening Session Patient Listening Session Summary March 15, 22, and 29, 2024
Long QT Syndrome (LQTS) EL-PFDD Meeting Host: SADS Foundation Meeting Report June 11, 2024
Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT) EL-PFDD Meeting Host: SADS Foundation Meeting Report June 11, 2024
Adult Dermatomyositis (DM) EL-PFDD Meeting Host: Myositis Support and Understanding Meeting Report June 7, 2024
Peroxisomal Disorders: Zellweger spectrum disorder (ZSD), acyl-CoA oxidase 1 deficiency (ACOX1), D-bifunctional protein deficiency (DPBD), and 2-methylacyl-CoA racemase deficiency (AMACR) EL-PFDD Meeting Host: The Global Foundation for Peroxisomal Disorders (GFPD) Meeting Report May 28, 2024
Rare, Autoimmune Peripheral Neuropathies: Guillain-Barre Syndrome (GBS), Multifocal Motor Neuropathy (MMN), and Anti-myeline-associated glycoprotein (Anti-MAG) neuropathy EL-PFDD Meeting Host: GBS|CIDP Foundation International Meeting Report May 16, 2024
Idiopathic Hypersomnia (IH) EL-PFDD Meeting Host: Sleep Consortium Meeting Report April 11, 2024
Congenital Cytomegalovirus Infection (cCMV) Patient Listening Session Patient Listening Session Summary April 4, 2024
Pelizaeus Merzbacher Disease (PMD) Patient Listening Session Patient Listening Session Summary August 22, 2023
Hypoparathyroidism EL-PFDD Meeting Host: HypoPARAthyroidism Association Meeting Report March 5, 2024
Dry Age-Related Macular Degeneration (Dry AMD) EL-PFDD Meeting Host: Foundation Fighting Blindness Meeting Report March 1, 2024
Pediatric Low-Grade Glioma (pLGG) EL-PFDD Meeting Host: Pediatric Brain Tumor Foundation Meeting Report February 23, 2024
GM2 gangliosidoses (Tay-Sachs and Sandhoff diseases) EL-PFDD Meeting Host: National Tay-Sachs & Allied Diseases Association (NTSAD) Meeting Report February 15, 2024
Immune Thrombotic Thrombocytopenic Purpura (iTTP) EL-PFDD Meeting Host: The Ree Wyn Foundation Meeting Report January 26, 2024
Pyruvate Dehydrogenase Complex Deficiency (PDCD) Patient Listening Session Patient Listening Session Summary September 8, 2023
Atypical Hemolytic Uremic Syndrome (aHUS) Patient Listening Session Patient Listening Session Summary September 21, 2023
Epidermolysis Bullosa (EB) EL-PFDD Meeting Host: Dystrophic Epidermolysis Bullosa Research Association of America (Debra of America) Meeting April 6, 2018
Spinal Cord Injury (SCI) Patient Listening Session Patient Listening Session Summary April 24, 2023
C. difficile Infections Patient Listening Session Patient Listening Session Summary March 10, 2023
Kidney Xenotransplantation EL-PFDD Meeting Host: National Kidney Foundation Meeting Report November 9, 2023
Polycystic Ovary Syndrome (PCOS) EL-PFDD Meeting Host: PCOS Challenge: The National Polycystic Ovary Syndrome Association Report pending November 3, 2023
Hypermobile Ehlers-Danlos Syndrome (hEDS) and Hypermobility Spectrum Disorders (HSD) EL-PFDD Meeting Host: The Ehlers-Danlos Society Report pending October 31, 2023
Classical Homocystinuria (HCU) EL-PFDD Meeting Host: HCU Network America Meeting Report October 27, 2023
STXBP1 EL-PFDD Meeting Host: STXBP1 Foundation Meeting Report October 20, 2023
Low-Grade Serous Ovarian Cancer (LGSOC) EL-PFDD Meeting Host: STAAR Ovarian Cancer Foundation Meeting Report October 13, 2023
Cystic Fibrosis (CF) EL-PFDD Meeting Host: Cystic Fibrosis Research Institute Meeting Report October 29, 2018
Spinocerebellar Ataxia Type 3 (SCA3) Patient Listening Session Patient Listening Session Summary September 22, 2023
Developmental and Epileptic Encephalopathy (DEE) EL-PFDD Meeting Host: Lennox-Gastaut Syndrome Foundation Meeting November 1, 2019
Cyclin-dependent kinase-like 5 deficiency disorder (CDKL5/CDD) EL-PFDD Meeting Host: Loulou Foundation and the International Foundation for CDKL5 Research (IFCR) Meeting Report November 1, 2019
Autosomal Recessive Polycystic Kidney Disease (ARPKD) EL-PFDD Meeting Host: PKD Foundation Meeting Report August 29, 2023
Carcinoid Syndrome Patient Listening Session Patient Listening Session Summary April 27, 2023
Canavan Disease Patient Listening Session Patient Listening Session Summary March 21, 2023
Post-finasteride Syndrome (PFS) Patient Listening Session Patient Listening Session Summary June 2, 2023
Prader-Willi Syndrome (PWS) EL-PFDD Meeting Host: PWSA|USA Meeting Report June 23, 2023
Arrhythmogenic Right Ventricular Cardiomyopathy/Dysplasia (ARVC/D) EL-PFDD Meeting Host: SADS Foundation Meeting Report June 20, 2023
Recurrent Clostridium difficile Infections (rCDI) Patient Listening Session Patient Listening Session Summary March 10, 2023
Sickle Cell Disease (SCD) Patient Listening Session Patient Listening Session Summary May 5, 2023
Pulmonary Alveolar Proteinosis (PAP) Patient Listening Session Patient Listening Session Summary July 8, 2020
Bronchopulmonary Dysplasia (BPD) Patient Listening Session Patient Listening Session Summary March 20, 2023
Juvenile Idiopathic Arthritis (JIA) EL-PFDD Meeting
Host: The Arthritis Foundation and the Childhood Arthritis and Rheumatology Research Alliance (CARRA)
Meeting Report April 2, 2018
Long COVID FDA-led PFDD Meeting • Agenda (English)
• Agenda (Spanish)
• Slides
• Recording (English)
• Recording (Spanish)
• Transcript
• Summary Report
April 25, 2023
Congenital Disorders of Glycosylation (CDG) Patient Listening Session Patient Listening Session Summary January 30, 2023
Juvenile Huntington’s Disease (JHD) Patient Listening Session Patient Listening Session Summary February 10, 2023
Pouchitis after a total colectomy and ideal pouch anal anastomosis (IPAA) surgery Patient Listening Session Patient Listening Session Summary January 14, 2023
SYNGAP1 EL-PFDD Meeting
Host: SYNGAP1 Foundation
Meeting Report November 19, 2020
Pre-Diagnosis Genetic Amyotrophic Lateral Sclerosis (ALS) and Frontotemporal Degeneration (FTD) Patient Listening Session Patient Listening Session Summary January 12, 2023
Proteus Syndrome Patient Listening Session Patient Listening Session Summary December 1, 2022
Stargardt Disease (STGD1) Patient Listening Session Patient Listening Session Summary June 24, 2022
Recurrent Respiratory Papillomatosis (RRP) Patient Listening Session Patient Listening Session Summary October 27, 2022
Wiskott-Aldrich Syndrome (WAS) and X-Linked Thrombocytopenia (XLT) EL-PFDD Meeting Host: Wiskott-Aldrich Foundation Meeting Report February 3, 2023
Autoimmune Hepatitis (AIH) EL-PFDD Meeting Host: Autoimmune Hepatitis Association Meeting January 27, 2023
Pemphigus and Pemphigoid EL-PFDD Meeting Host: International Pemphigus and Pemphigoid Foundation Meeting Report January 25, 2023
Cerebral Creatine Deficiency Syndromes (CCDS): L-arginine:glycine amidinotransferase (AGAT) deficiency, guanidinoacetate methyltransferase (GAMT) deficiency, and creatine transporter deficiency (CTD) EL-PFDD Meeting Host: Association for Creatine Deficiencies Meeting Report January 24, 2023
Narcolepsy and Idiopathic Hypersomnia (IH) Patient Listening Session Patient Listening Session Summary August 8, 2022
Hypophosphatasia (HPP) EL-PFDD Meeting Host: Soft Bones, Inc., The US Hypophosphatasia Foundation Meeting Report November 15, 2022
GM1 gangliosidosis EL-PFDD Meeting Host: Cure GM1 Foundation Meeting Report October 14, 2022
Metachromatic Leukodystrophy (MLD) EL-PFDD Meeting Host: Cure MLD, The Calliope Joy Foundation, MLD Foundation, The United Leukodystrophy Foundation, and the Global Leukodystrophy Initiative Meeting Report October 21, 2022
Schizophrenia EL-PFDD Meeting Host: The American Foundation for Suicide Prevention, Mental Health America, the National Alliance on Mental Illness (NAMI), the National Council for Mental Wellbeing, and Schizophrenia and Psychosis Action Alliance (S&PAA) Meeting Report November 2, 2022
Phelan-McDermid Syndrome (PMS) EL-PFDD Meeting Host: CureSHANK Meeting Report November 8, 2022
Kennedy’s Disease (KD)/ Spinal and bulbar muscular atrophy (SBMA) EL-PFDD Meeting Host: Kennedy’s Disease Association Meeting Report November 9, 2022
Short Bowel Syndrome (SBS) Patient Listening Session Patient Listening Session Summary July 19, 2022
Huntington’s Disease (HD) Pre-Symptomatic Population Patient Listening Session Patient Listening Session Summary July 25, 2022
Spinal Muscular Atrophy (SMA) Patient Listening Session Patient Listening Session Summary August 4, 2022
Alstrom Syndrome (AS) EL-PFDD Meeting
Host: Alstrom International
Meeting Report September 22, 2022
Reducing Cardiac Late Effects in Pediatric Cancer Survivors EL-PFDD Meeting
Host: Children’s Cancer Cause
Meeting Report September 15, 2022
Fabry Disease EL-PFDD Meeting
Host: National Kidney Foundation and Fabry Support & Information Group
Meeting Report September 19, 2022
Galactosemia EL-PFDD Meeting
Host: National Organization for Rare Disorders (NORD)
Meeting Report September 1, 2022
Limb-Girdle Muscular Dystrophies (LGMD) EL-PFDD Meeting
Host: Coalition to Cure Calpain 3, CureLGMD2i, the Kurt+Peter Foundation, the LGMD2D Foundation, the McColl-Lockwood Laboratory for Muscular Dystrophy Research, and the Speak Foundation
Meeting Report September 23, 2022
Primary Biliary Cholangitis (PBC) EL-PFDD Meeting Host: Global Liver Institute Meeting Report February 4, 2022
Food Allergies EL-PFDD Meeting Host: Food Allergy Research and Education (FARE) Meeting Report September 9, 2021
Asthma EL-PFDD Meeting Host: Asthma and Allergy Foundation of America (AAFA) Meeting Report September 20, 2021
Myotubular and Centronuclear Myopathy (MTM/CNM) EL-PFDD Meeting Host: MTM-CNM Family Connection and Where There’s A Will There’s A Cure Meeting August 3, 2021
Rett Syndrome EL-PFDD Meeting Host: Rett Syndrome Research Trust (RSRT) Meeting Report March 11, 2022
Membranous Nephropathy (MN) EL-PFDD Meeting Host: National Kidney Foundation and NephCure Kidney International Meeting Report August 27, 2021
Cancer Cachexia EL-PFDD Meeting Host: Cancer Support Community Meeting Report November 2, 2021
Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) EL-PFDD Meeting Host: GBS|CIDP Foundation International Meeting Report March 25, 2022
Primary Hyperoxaluria (PH) EL-PFDD Meeting Host: The Oxalosis and Hyperoxaluria Foundation Meeting Report October 5, 2020
Post-Transplant Lymphoproliferative Disorder (PTLD) EL-PFDD Meeting Host: National Organization for Rare Disorders (NORD) Meeting Report May 4, 2022
Bronchiolitis Obliterans Syndrome (BOS) EL-PFDD Meeting Host: Lung Transplant Foundation Meeting Report June 22, 2022
Succinic Semialdehyde Dehydrogenase Deficiency (SSADHD) EL-PFDD Meeting Host: SSADH Association Meeting Report July 8, 2022
X-linked Retinitis Pigmentosa (XLRP) EL-PFDD Meeting Host: Fighting Blindness Foundation Meeting Report June 7, 2022
Congenital Muscular Dystrophy (CMD) EL-PFDD Meeting Host: Cure CMD Report unavailable July 1, 2022
Hermansky-Pudlak Syndrome (HPS) EL-PFDD Meeting Host: Hermansky-Pudlak Syndrome Network Meeting Report June 10, 2022
X-linked Adrenoleukodystrophy (ALD) EL-PFDD Meeting Host: ALD Connect Meeting Report July 22, 2022
Acromegaly
EL-PFDD Meeting Host: Acromegaly Community, Inc. Meeting Report January 21, 2021
Acute Porphyrias EL-PFDD Meeting Host: American Porphyria Foundation Meeting Report March 1, 2017
Adrenomyeloneuropathy (AMN) Patient Listening Session Patient Listening Session Summary May 7, 2021
Adult Dermatomyositis (DM) Patient Listening Session Patient Listening Session Summary April 26, 2022
Adult Polyglucosan Body Disease (APBD) Patient Listening Session Patient Listening Session Summary October 28, 2021
Alopecia Areata FDA-led PFDD Meeting • Agenda
• Slides
• Recordings (Part 1, Part 2)
• Transcript
• Summary Report
September 11, 2017
Alpha-1 Antitrypsin Deficiency (AATD) FDA-led PFDD Meeting • Agenda
• Slides
• Recordings (Part 1, Part 2)
• Transcript
• Summary Report
September 29, 2015
Alport Syndrome (AS) EL-PFDD Meeting Host: National Kidney Foundation and the Alport Syndrome Foundation Meeting Report August 3, 2018
Amyloidosis EL-PFDD Meeting Host: The Amyloidosis Research Consortium Meeting Report November 16, 2015
Amyotrophic Lateral Sclerosis (ALS) N/A IMPACT ALS Survey Report N/A
Angelman Syndrome EL-PFDD Meeting Host: Foundation for Angelman Syndrome Therapeutics (FAST) and the Angelman Syndrome Foundation (ASF) Meeting Report April 7, 2025
Autism (ASD) FDA-led PFDD Meeting • Agenda
• Slides
• Recordings (Part 1, Part 2)
• Transcript
• Summary Report
May 4, 2017
Autoimmune Pulmonary Alveolar Proteinosis (aPAP) EL-PFDD Meeting Host: National Organization for Rare Disorders (NORD) Report Pending May 7, 2025
Barth Syndrome (BTHS) EL-PFDD Meeting Host: The Barth Syndrome Foundation Meeting Report July 18, 2018
Breast Cancer FDA-led PFDD Meeting • Agenda
• Slides
• Recordings (Part 1, Part 2)
• Transcript
• Summary Report
April 2, 2015
Celiac Disease Patient Listening Session Patient Listening Session Summary February 20, 2019
Cerebral Cavernous Malformation (CCM) Patient Listening Session Patient Listening Session Summary November 6, 2019
Cerebrotendinous Xanthomatosis (CTX) EL-PFDD Meeting Host: United Leukodystrophy Foundation of America Meeting Report September 14, 2021
Chagas Disease FDA-led PFDD Meeting • Agenda
• Slides
• Recordings (Part 1, Part 2, Part 3, Part 4)
• Transcript
• Summary Report
April 28, 2015
Charcot-Marie-Tooth and Inherited Neuropathies (CMT/IN) EL-PFDD Meeting Host: Hereditary Neuropathy Foundation Meeting Report September 28, 2018
Chemotherapy-Induced Hearing Loss EL-PFDD Meeting Host: Children’s Cause for Cancer Advocacy, Children’s Brain Tumor Foundation, Mattie Miracle Cancer Foundation, and Momcology Meeting Report September 13, 2018
Childhood Cerebral Adrenal Leukodystrophy (CCALD) Patient Listening Session Patient Listening Session Summary November 13, 2019
Chronic Fatigue Syndrome/Myalgic Encephalomyelitis (CFS & ME) FDA-led PFDD Meeting • Agenda
• Slides (Part 1, Part 2)
• Recordings (Part 1, Part 2, Part 3, Part 4, Part 5, Part 6, Part 7, Part 8)
• Transcript
• Summary Report
April 25, 2013
Chronic Pain FDA-led PFDD Meeting • Agenda
• Slides
• Recordings (Part 1, Part 2)
• Transcript
• Summary Report
July 9, 2018
Complement 3 Glomerulopathy (C3G) EL-PFDD Meeting Host: National Kidney Foundation Meeting Report August 4, 2017
Cystic Fibrosis (CF) – nonsense mutations Patient Listening Session Patient Listening Session Summary July 15, 2021
Atopic Dermatitis (Eczema) EL-PFDD Meeting Host: Allergy and Asthma Network, Asthma and Allergy Foundation of America, Global Parents for Eczema Research, International Topical Steroid Awareness Network, and National Eczema Association Meeting Report September 23, 2019
Dravet Syndrome (DS) EL-PFDD Meeting Host: The Dravet Syndrome Foundation Meeting Report February 3, 2022
Fabry Disease Patient Listening Session Patient Listening Session Summary December 4, 2018
Facioscapulohumeral Muscular Dystrophy (FSHD) EL-PFDD Meeting Host: FSHD Society Meeting Report June 29, 2020
Female Sexual Dysfunction (FSD) FDA-led PFDD Meeting • Agenda (Day 1, Day 2)
• Slides (Day 1)
• Recordings (Part 1, Part 2)
• Transcript (Day 1, Day 2)
• Summary Report
October 27, 2014
Fibrodysplasia Ossificans Progressiva (FOP) Patient Listening Session Patient Listening Session Summary May 29, 2019
Fibromyalgia FDA-led PFDD Meeting • Agenda
• Slides
• Recordings (Part 1, Part 2)
• Transcript
• Summary Report
March 26, 2014
Focal Segmental Glomerulosclerosis (FSGS) EL-PFDD Meeting Host: National Kidney Foundation and NephCure Kidney International Meeting Report August 28, 2020
Fragile X Syndrome (FXS) EL-PFDD Meeting Host: National Fragile X Foundation Meeting Report March 3, 2021
Friedreich’s Ataxia (FA) EL-PFDD Meeting Host: Friedreich’s Ataxia Research Alliance Meeting Report June 2, 2017
Frontotemporal Degeneration (FTD) EL-PFDD Meeting Host: Association for Frontotemporal Degeneration (AFTD) Meeting Report March 5, 2021
Functional Gastrointestinal (GI) Disorders FDA-led PFDD Meeting • Agenda
• Slides
• Recordings (Part 1, Part 2)
• Transcript
• Summary Report
May 11, 2015
Gastroparesis Patient Listening Session Patient Listening Session Summary December 2, 2019
Gene Therapy as a Treatment Modality for Hemophilia Patient Listening Session Patient Listening Session Summary October 23, 2018
Glycogen Storage Disease (GSD) Type 1 – Adult Patients Patient Listening Session Patient Listening Session Summary March 18, 2021
Glycogen Storage Disease (GSD) Type 1 – Caregivers to Pediatric Patients Patient Listening Session Patient Listening Session Summary March 25, 2021
Glycogen Storage Disease (GSD) Type 1B Patient Listening Session Patient Listening Session Summary March 3, 2022
Gorlin Syndrome (GS) Patient Listening Session Patient Listening Session Summary November 9, 2020
Gorlin Syndrome (GS) EL-PFDD Meeting Host: Gorlin Syndrome Alliance Meeting Report October 8, 2021
Guillain-Barre’ Syndrome (GBS) Patient Listening Session Patient Listening Session Summary September 29, 2020
Hemophilia A, Hemophilia B, von Willebrand Disease (VWB), and Other Heritable Bleeding Disorders FDA-led PFDD Meeting • Agenda
• Slides
• Recordings (Part 1, Part 2)
• Transcript
• Summary Report
September 22, 2014
Chronic Hepatitis B EL-PFDD Meeting Host: Hepatitis B Foundation Meeting Report June 9, 2020
Hereditary Angioedema (HAE) FDA-led PFDD Meeting • Agenda
• Recordings (Part 1, Part 2)
• Transcript
• Summary Report
September 25, 2017
Homocystinuria (HCU) Patient Listening Session Patient Listening Session Summary June 26, 2020
Human Immunodeficiency Virus (HIV) FDA-led PFDD Meeting • Agenda
• Slides
• Recordings (Part 1, Part 2, Part 3, Part 4)
• Transcript
• Summary Report
June 14, 2013
Hunter Syndrome (MPS II) Patient Listening Session Patient Listening Session Summary February 4, 2020
Hunter Syndrome (MPS II) N/A Toileting Ability Survey Report N/A
Huntington’s Disease (HD) FDA-led PFDD Meeting • Agenda
• Slides
• Recordings (Part 1, Part 2, Part 3, Part 4)
• Transcript
• Summary Report
September 22, 2015
Hypereosinophilic Syndromes (HES) EL-PFDD Meeting Host: American Partnership for Eosinophilic Disorders Meeting Report March 23, 2018
Hyperhidrosis (Hh) EL-PFDD Meeting Host: International Hyperhidrosis Society Meeting Report November 13, 2017
Hypertrophic Cardiomyopathy (HCM) EL-PFDD Meeting Host: Hypertrophic Cardiomyopathy Association Meeting Report June 26, 2020
Hypomyelination with Atrophy of Basal Ganglia and Cerebellum (H-ABC) Patient Listening Session Patient Listening Session Summary May 9, 2022
Hypophosphatemia EL-PFDD Meeting Host: XLH Network Meeting Report October 5, 2018
Hypothalamic Obesity (HO) Patient Listening Session Patient Listening Session Summary October 22, 2021
Ichthyosis Patient Listening Session Patient Listening Session Summary September 17, 2021
Idiopathic Pulmonary Fibrosis (IPF) FDA-led PFDD Meeting • Agenda
• Slides
• Recordings (Part 1, Part 2)
• Transcript
• Summary Report
September 26, 2014
IgA Nephropathy (IgAN) EL-PFDD Meeting Host: National Kidney Foundation and the IgA Nephropathy Foundation of America Meeting Report August 19, 2019
Immune Thrombocytopenia (ITP) EL-PFDD Meeting Host: Platelet Disorder Support Association Meeting Report July 26, 2019
Inborn Errors of Metabolism (IEM) FDA-led PFDD Meeting • Agenda
• Slides
• Recordings (Part 1, Part 2)
• Transcript
• Summary Report
June 10, 2014
Inclusion Body Myositis (IBM) Patient Listening Session Patient Listening Session Summary March 5, 2020
Krabbe Disease EL-PFDD Meeting Host: National Organization for Rare Disorders (NORD) Meeting Report October 29, 2020
Late Onset GM2 gangliosidoses (Tay-Sachs and Sandhoff Diseases) Patient Listening Session Patient Listening Session Summary January 15, 2021
Limb-Girdle Muscular Dystrophies (LGMD) Patient Listening Session Patient Listening Session Summary October 20, 2020
Lung Cancer FDA-led PFDD Meeting • Agenda
• Slides
• Recordings (Part 1, Part 2)
• Transcript
• Summary Report
June 28, 2013
Lupus – Diversity in Clinical Trials Patient Listening Session • Patient Listening Session Summary – Session 1
• Patient Listening Session Summary – Session 2
• Patient Listening Session Summary – Session 3
March 31, 2021 – Session 1
April 2, 2021 – Session 2
April 6, 2021 – Session 3
Lupus EL-PFDD Meeting Host: Lupus and Allied Diseases Association, Lupus Foundation of America, and the Lupus Research Alliance Meeting Report September 25, 2017
Major Depressive Disorder (MDD) EL-PFDD Meeting Host: Depression and Bipolar Support Alliance Meeting Report November 16, 2018
Mastocytosis Patient Listening Session Patient Listening Session Summary September 28, 2021
Mitochondrial Disease EL-PFDD Meeting Host: United Mitochondrial Disease Foundation Meeting Report March 29, 2019
Myeloproliferative Neoplasms (MPNs) EL-PFDD Meeting Host: MPN Research Foundation Meeting Report September 16, 2019
Myotonic Dystrophy (DM) EL-PFDD Meeting Host: Myotonic Dystrophy Foundation Meeting Report September 15, 2016
Narcolepsy FDA-led PFDD Meeting • Agenda
• Slides
• Recordings (Part 1, Part 2)
• Transcript
• Summary Report
September 24, 2013
Neurofibromatosis (NF) Patient Listening Session Patient Listening Session Summary June 13, 2019
Neuronopathic Gaucher Disease (nGD) (Types 2 & 3) Patient Listening Session Patient Listening Session Summary September 9, 2021
Neuropathic Pain Associated with Peripheral Neuropathy FDA-led PFDD Meeting • Agenda
• Slides
• Recordings (Part 1, Part 2, Part 3)
• Transcript
• Summary Report
June 10, 2016
Niemann-Pick Type C (NPC) EL-PFDD Meeting Host: Ara Parseghian Medical Research Fund at Notre Dame, Hide & Seek Foundation, Dana's Angels Research Trust, Hope for Marian, National Niemann-Pick Disease Foundation, Niemann-Pick Canada, Firefly Fund, and Johnathon's Dreams Meeting Report March 18, 2019
Nonalcoholic Steatohepatitis (NASH) EL-PFDD Meeting Host: Global Liver Institute Meeting Report November 4, 2021
Non-Tuberculous Mycobacterial (NTM) Lung Infections FDA-led PFDD Meeting • Agenda
• Slides
• Recordings (Part 1, Part 2, Part 3, Part 4)
• Transcript
• Summary Report
October 15, 2015
Obstructive Sleep Apnea (OSA) EL-PFDD Meeting Host: American Sleep Apnea Association Meeting Report June 8, 2018
Ocular Melanoma (OM) Patient Listening Session Patient Listening Session Summary January 27, 2020
Opioid Use Disorder (OUD) FDA-led PFDD Meeting • Agenda
• Slides
• Recordings (Part 1, Part 2, Part 3)
The audio recording of this meeting is publicly available using the links above. Because of the sensitive nature of the meeting topic, and the importance of gathering candid, meaningful input from individuals who have come forward to speak about living with opioid use disorder, there is no video recording of the meeting.
• Transcript
• Summary Report
April 17, 2018
Osteoarthritis (OA) Patient Listening Session Patient Listening Session Summary August 7, 2019
Osteoarthritis (OA) EL-PFDD Meeting Host: Arthritis Foundation Meeting Report March 8, 2017
Osteogenesis Imperfecta (OI) Patient Listening Session Patient Listening Session Summary September 17, 2019
Pachyonychia Congenita (PC) EL-PFDD Meeting Host: Pachyonychia Congenita Project Meeting Report April 6, 2018
Pancreatitis EL-PFDD Meeting Host: National Pancreas Foundation and FCS Foundation Meeting Report March 3, 2020
Parkinson’s Disease FDA-led PFDD Meeting • Agenda
• Slides
• Recordings (Part 1, Part 2, Part 3, Part 4)
• Transcript
• Summary Report
September 22, 2015
Patients Who Have Received an Organ Transplant FDA-led PFDD Meeting • Agenda
• Slides
• Recordings (Part 1, Part 2, Part 3, Part 4)
• Transcript
• Summary Report
September 27, 2016
Pemphigus and Pemphigoid Patient Listening Session Patient Listening Session Summary February 8, 2021
Polyglutamine Spinocerebellar Ataxias (SCAs) and Dentatorubal-Pallidoluysian Atrophy (DRPLA) EL-PFDD Meeting Host: National Ataxia Foundation and CureDRPLA Meeting Report September 25, 2020
Pompe Disease EL-PFDD Meeting Host: Muscular Dystrophy Association Meeting Report July 13, 2020
Primary Sclerosing Cholangitis (PSC) EL-PFDD Meeting Host: PSC Partners Seeking a Cure Meeting Report October 23, 2020
Progressive Multifocal Leukoencephalopathy (PML) Patient Listening Session • Patient Listening Session Summary – Session 1
• Patient Listening Session Summary – Session 2
July 22, 2020 – Session 1
September 11, 2020 – Session 2
Psoriasis FDA-led PFDD Meeting • Agenda
• Slides
• Recordings (Part 1, Part 2)
• Transcript
• Summary Report
March 17, 2016
Pulmonary Arterial Hypertension (PAH) FDA-led PFDD Meeting • Agenda
• Slides
• Recordings (Part 1, Part 2)
• Transcript
• Summary Report
May 13, 2014
Pulmonary Sarcoidosis Patient Listening Session Patient Listening Session Summary April 21, 2022
Pyruvate Kinase Deficiency EL-PFDD Meeting Host: National Organization for Rare Disorders (NORD) Meeting Report September 20, 2019
Sanfilippo Syndrome Patient Listening Session Patient Listening Session Summary May 13, 2019
Sanfilippo Syndrome – Pediatric Patient Listening Session Patient Listening Session Summary October 17, 2019
Sarcopenia FDA-led PFDD Meeting • Agenda
• Slides
• Recording (Full recording)
• Transcript
• Summary Report
April 6, 2017
SCN2A Related Disorders Patient Listening Session Patient Listening Session Summary April 28, 2021
Sensorineural Hearing Loss (SNHL) EL-PFDD Meeting Host: Hearing Loss Association of America Meeting Report May 25, 2021
Sickle Cell Disease (SCD) FDA-led PFDD Meeting • Agenda
• Slides
• Recordings (Part 1, Part 2)
• Transcript
• Summary Report
February 7, 2014
Smith-Magenis Syndrome (SMS) Patient Listening Session Patient Listening Session Summary August 12, 2020
Spinal Muscular Atrophy (SMA) EL-PFDD Meeting Host: Cure SMA Meeting Report April 18, 2017
Stimulant Use Disorder FDA-led PFDD Meeting • Agenda
• Slides
• Recording
• Transcript
• Summary Report
October 6, 2020
Systemic Sclerosis FDA-led PFDD Meeting • Agenda
• Slides
• Recording
• Transcript
• Summary Report
October 13, 2020
Thymidine Kinase 2 Deficiency (TK2d) Patient Listening Session Patient Listening Session Summary January 31, 2022
Tuberous Sclerosis Complex (TSC) EL-PFDD Meeting Host: Tuberous Sclerosis Alliance Meeting Report June 21, 2017
Valosin Containing Protein Disease (VCP) Patient Listening Session Patient Listening Session Summary May 5, 2020
Vascular Ehlers-Danlos Syndrome (VEDS) Patient Listening Session Patient Listening Session Summary October 7, 2020
Vitiligo FDA-led PFDD Meeting • Agenda
• Slides
• Recording
• Transcript
• Summary Report
March 8, 2021
Von Hippel Lindau (VHL) Patient Listening Session Patient Listening Session Summary June 11, 2020
Xerostomia EL-PFDD Meeting Host: Head and Neck Cancer Alliance (HNCA), the National Foundation of Swallowing Disorders (NFOSD), Support for People with Oral and Head and Neck Cancer (SPOHNC), and the THANC (Thyroid, Head and Neck Cancer) Foundation Meeting Report August 19, 2021
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Reproduced from FDA Drugs (Whats New) · view original · public domain or open-licensed government work