Condition-Specific Meeting Reports and Other Information Related to Patients' Experience
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 Other information includes proposed draft guidance relating to patient experience data, natural history studies, or other condition-specific background on condition and discussion of unmet medical need.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.
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| Disease or Condition (alphabetical) | Type of Meeting | Resource(s) | Meeting Date |
|---|---|---|---|
| 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) | Report pending | 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 | Report pending | 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) | Report pending | 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 | Report pending | 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) | Report pending | 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 | Report pending | 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 | Report pending | 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 | 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 | 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 | Report pending | 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 pending | 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 |
| September 11, 2017 |
| Alpha-1 Antitrypsin Deficiency (AATD) | FDA-led PFDD Meeting |
| 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) | Report Pending | April 7, 2025 |
| Autism (ASD) | FDA-led PFDD Meeting |
| 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 |
| 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 |
| 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 |
| April 25, 2013 |
| Chronic Pain | FDA-led PFDD Meeting |
| 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 |
| October 27, 2014 |
| Fibrodysplasia Ossificans Progressiva (FOP) | Patient Listening Session | Patient Listening Session Summary | May 29, 2019 |
| Fibromyalgia | FDA-led PFDD Meeting |
| 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 |
| 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 |
| 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 |
| September 25, 2017 |
| Homocystinuria (HCU) | Patient Listening Session | Patient Listening Session Summary | June 26, 2020 |
| Human Immunodeficiency Virus (HIV) | FDA-led PFDD Meeting |
| 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 |
| 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 |
| 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 |
| 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 |
| June 28, 2013 |
| Lupus – Diversity in Clinical Trials | Patient Listening Session | 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 |
| 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 |
| 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 |
| 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 | 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.
| 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 |
| September 22, 2015 |
| Patients Who Have Received an Organ Transplant | FDA-led PFDD Meeting |
| 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 | July 22, 2020 – Session 1 September 11, 2020 – Session 2 | |
| Psoriasis | FDA-led PFDD Meeting |
| March 17, 2016 |
| Pulmonary Arterial Hypertension (PAH) | FDA-led PFDD Meeting |
| 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 |
| 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 | FDA-led PFDD Meeting |
| 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 |
| October 6, 2020 |
| Systemic Sclerosis | FDA-led PFDD Meeting |
| 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 |
| 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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