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 Other information includes proposed draft guidance relating to patient experience data, natural history studies, or other condition-specific background on condition and discussion of unmet medical need.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 Patient Affairs 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 Patient Affairs.
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 |
---|---|---|---|
Acromegaly
| EL-PFDD Meeting Host: Acromegaly Community, Inc. | Meeting ReportExternal Link Disclaimer | January 21, 2021 |
Acute Porphyrias | EL-PFDD Meeting Host: American Porphyria Foundation | Meeting ReportExternal Link Disclaimer | March 1, 2017 |
Adrenomyeloneuropathy (AMN) | Patient Listening Session | Patient Listening Session Summary | May 7, 2021 |
Adult Dermatomyositis (DM) | EL-PFDD Meeting Host: Myositis Support and Understanding | Report pending | June 7, 2024 |
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 |
Adult-onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP) | Patient Listening Session | Patient Listening Session | January 17, 2024 |
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 ReportExternal Link Disclaimer | August 3, 2018 |
Alstrom Syndrome (AS) | EL-PFDD Meeting Host: Alstrom International | Meeting ReportExternal Link Disclaimer | September 22, 2022 |
Amyloidosis | EL-PFDD Meeting Host: The Amyloidosis Research Consortium | Meeting ReportExternal Link Disclaimer | November 16, 2015 |
Amyotrophic Lateral Sclerosis (ALS) | N/A | IMPACT ALS Survey ReportExternal Link Disclaimer | N/A |
Arrhythmogenic Right Ventricular Cardiomyopathy/Dysplasia (ARVC/D) | EL-PFDD Meeting Host: SADS Foundation | Meeting ReportExternal Link Disclaimer | June 20, 2023 |
Asthma | EL-PFDD Meeting Host: Asthma and Allergy Foundation of America (AAFA) | Meeting ReportExternal Link Disclaimer | September 20, 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 ReportExternal Link Disclaimer | September 23, 2019 |
Attention-Deficit/Hyperactivity Disorder (ADHD) | Patient Listening Session | Patient Listening Session Summary | March 15, 22, and 29, 2024 |
Atypical Hemolytic Uremic Syndrome (aHUS) | Patient Listening Session | Patient Listening Session Summary | September 21, 2023 |
Autism (ASD) | FDA-led PFDD Meeting |
| May 4, 2017 |
Autoimmune Hepatitis (AIH) | EL-PFDD Meeting Host: Autoimmune Hepatitis Association | MeetingExternal Link Disclaimer | January 27, 2023 |
Autosomal Recessive Polycystic Kidney Disease (ARPKD) | EL-PFDD Meeting Host: PKD Foundation | Meeting ReportExternal Link Disclaimer | August 29, 2023 |
Barth Syndrome (BTHS) | EL-PFDD Meeting Host: The Barth Syndrome Foundation | Meeting ReportExternal Link Disclaimer | July 18, 2018 |
Breast Cancer | FDA-led PFDD Meeting |
| April 2, 2015 |
Bronchiolitis Obliterans Syndrome (BOS) | EL-PFDD Meeting Host: Lung Transplant Foundation | Meeting ReportExternal Link Disclaimer | June 22, 2022 |
Bronchopulmonary Dysplasia (BPD) | Patient Listening Session | Patient Listening Session Summary | March 20, 2023 |
C. difficile Infections | Patient Listening Session | Patient Listening Session Summary | March 10, 2023 |
Canavan Disease | Patient Listening Session | Patient Listening Session Summary | March 21, 2023 |
Cancer Cachexia | EL-PFDD Meeting Host: Cancer Support Community | Meeting ReportExternal Link Disclaimer | November 2, 2021 |
Carcinoid Syndrome | Patient Listening Session | Patient Listening Session Summary | April 27, 2023 |
Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT) | EL-PFDD Meeting Host: SADS Foundation | Meeting ReportExternal Link Disclaimer | June 11, 2024 |
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 |
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 ReportExternal Link Disclaimer | January 24, 2023 |
Cerebrotendinous Xanthomatosis (CTX) | EL-PFDD Meeting Host: United Leukodystrophy Foundation of America | Meeting ReportExternal Link Disclaimer | 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 ReportExternal Link Disclaimer | 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 ReportExternal Link Disclaimer | 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 Hepatitis B | EL-PFDD Meeting Host: Hepatitis B Foundation | Meeting ReportExternal Link Disclaimer | June 9, 2020 |
Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) | EL-PFDD Meeting Host: GBS|CIDP Foundation International | Meeting ReportExternal Link Disclaimer | March 25, 2022 |
Chronic Pain | FDA-led PFDD Meeting |
| July 9, 2018 |
Classic Congenital Adrenal Hyperplasia (CAH) | EL-PFDD Meeting Host: CARES Foundation | Report pending | October 1, 2024 |
Classical Homocystinuria (HCU) | EL-PFDD Meeting Host: HCU Network America | Meeting ReportExternal Link Disclaimer | October 27, 2023 |
Complement 3 Glomerulopathy (C3G) | EL-PFDD Meeting Host: National Kidney Foundation | Meeting ReportExternal Link Disclaimer | August 4, 2017 |
Congenital Cytomegalovirus Infection (cCMV) | Patient Listening Session | Patient Listening Session Summary | April 4, 2024 |
Congenital Disorders of Glycosylation (CDG) | Patient Listening Session | Patient Listening Session Summary | January 30, 2023 |
Congenital Muscular Dystrophy (CMD) | EL-PFDD Meeting Host: Cure CMD | Report pending | July 1, 2022 |
Cyclin-dependent kinase-like 5 deficiency disorder (CDKL5/CDD) | EL-PFDD Meeting Host: Loulou Foundation and the International Foundation for CDKL5 Research (IFCR) | Meeting ReportExternal Link Disclaimer | November 1, 2019 |
Cystic Fibrosis (CF) | EL-PFDD Meeting Host: Cystic Fibrosis Research Institute | Meeting ReportExternal Link Disclaimer | October 29, 2018 |
Cystic Fibrosis (CF) – nonsense mutations | Patient Listening Session | Patient Listening Session Summary | July 15, 2021 |
Dementia with Lewy Bodies (DLB) | EL-PFDD Meeting Host: Lewy Body Dementia Association | Report pending | October 15, 2024 |
Developmental and Epileptic Encephalopathy (DEE) | EL-PFDD Meeting Host: Lennox-Gastaut Syndrome Foundation | MeetingExternal Link Disclaimer | November 1, 2019 |
Dravet Syndrome (DS) | EL-PFDD Meeting Host: The Dravet Syndrome Foundation | Meeting ReportExternal Link Disclaimer | February 3, 2022 |
Dry Age-Related Macular Degeneration (Dry AMD) | EL-PFDD Meeting Host: Foundation Fighting Blindness | Report pending | March 1, 2024 |
Epidermolysis Bullosa (EB) | EL-PFDD Meeting Host: Dystrophic Epidermolysis Bullosa Research Association of America (Debra of America) | MeetingExternal Link Disclaimer | April 6, 2018 |
Fabry Disease | EL-PFDD Meeting Host: National Kidney Foundation and Fabry Support & Information Group | Report pending | September 19, 2022 |
Fabry Disease | Patient Listening Session | Patient Listening Session Summary | December 4, 2018 |
Facioscapulohumeral Muscular Dystrophy (FSHD) | EL-PFDD Meeting Host: FSHD Society | Meeting ReportExternal Link Disclaimer | 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 ReportExternal Link Disclaimer | August 28, 2020 |
Food Allergies | EL-PFDD Meeting Host: Food Allergy Research and Education (FARE) | Meeting ReportExternal Link Disclaimer | September 9, 2021 |
Fragile X Syndrome (FXS) | EL-PFDD Meeting Host: National Fragile X Foundation | Meeting ReportExternal Link Disclaimer | March 3, 2021 |
Friedreich’s Ataxia (FA) | EL-PFDD Meeting Host: Friedreich’s Ataxia Research Alliance | Meeting ReportExternal Link Disclaimer | June 2, 2017 |
Frontotemporal Degeneration (FTD) | EL-PFDD Meeting Host: Association for Frontotemporal Degeneration (AFTD) | Meeting ReportExternal Link Disclaimer | March 5, 2021 |
Functional Gastrointestinal (GI) Disorders | FDA-led PFDD Meeting |
| May 11, 2015 |
Galactosemia | EL-PFDD Meeting Host: National Organization for Rare Disorders (NORD) | Meeting ReportExternal Link Disclaimer | September 1, 2022 |
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 |
GM1 gangliosidosis | EL-PFDD Meeting Host: Cure GM1 Foundation | Meeting ReportExternal Link Disclaimer | October 14, 2022 |
GM2 gangliosidoses (Tay-Sachs and Sandhoff diseases) | EL-PFDD Meeting Host: National Tay-Sachs & Allied Diseases Association (NTSAD) | Meeting ReportExternal Link Disclaimer | February 15, 2024 |
Gorlin Syndrome (GS) | Patient Listening Session | Patient Listening Session Summary | November 9, 2020 |
Gorlin Syndrome (GS) | EL-PFDD Meeting Host: Gorlin Syndrome Alliance | Meeting ReportExternal Link Disclaimer | October 8, 2021 |
Guillain-Barre’ Syndrome (GBS) | Patient Listening Session | Patient Listening Session Summary | September 29, 2020 |
Healthcare Challenges and Unmet Medical Needs of Transgender Adolescents | Patient Listening Session | Patient Listening Session Summary | June 29, 2021 |
Healthcare Challenges and Unmet Medical Needs of Transgender Adults | Patient Listening Session | Patient Listening Session Summary | June 1, 2021 |
Hemophilia A, Hemophilia B, von Willebrand Disease (VWB), and Other Heritable Bleeding Disorders | FDA-led PFDD Meeting |
| September 22, 2014 |
Hereditary Angioedema (HAE) | FDA-led PFDD Meeting |
| September 25, 2017 |
Hermansky-Pudlak Syndrome (HPS) | EL-PFDD Meeting Host: Hermansky-Pudlak Syndrome Network | Meeting ReportExternal Link Disclaimer | June 10, 2022 |
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 ReportExternal Link Disclaimer | N/A |
Huntington’s Disease (HD) | FDA-led PFDD Meeting |
| September 22, 2015 |
Huntington’s Disease (HD) Pre-Symptomatic Population | Patient Listening Session | Patient Listening Session Summary | July 25, 2022 |
Hypereosinophilic Syndromes (HES) | EL-PFDD Meeting Host: American Partnership for Eosinophilic Disorders | Meeting ReportExternal Link Disclaimer | March 23, 2018 |
Hyperhidrosis (Hh) | EL-PFDD Meeting Host: International Hyperhidrosis Society | Meeting ReportExternal Link Disclaimer | November 13, 2017 |
Hypermobile Ehlers-Danlos Syndrome (hEDS) and Hypermobility spectrum disorders (HSD) | EL-PFDD Meeting Host: The Ehlers-Danlos Society | Report pending | October 31, 2023 |
Hypertrophic Cardiomyopathy (HCM) | EL-PFDD Meeting Host: Hypertrophic Cardiomyopathy Association | Meeting ReportExternal Link Disclaimer | June 26, 2020 |
Hypomyelination with Atrophy of Basal Ganglia and Cerebellum (H-ABC) | Patient Listening Session | Patient Listening Session Summary | May 9, 2022 |
Hypoparathyroidism | EL-PFDD Meeting Host: HypoPARAthyroidism Association | Meeting ReportExternal Link Disclaimer | March 5, 2024 |
Hypophosphatasia (HPP) | EL-PFDD Meeting Host: Soft Bones, Inc., The US Hypophosphatasia Foundation | Meeting ReportExternal Link Disclaimer | November 15, 2022 |
Hypophosphatemia | EL-PFDD Meeting Host: XLH Network | Meeting ReportExternal Link Disclaimer | 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 Hypersomnia (IH) | EL-PFDD Meeting Host: Sleep Consortium | Meeting ReportExternal Link Disclaimer | April 11, 2024 |
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 ReportExternal Link Disclaimer | August 19, 2019 |
Immune Thrombocytopenia (ITP) | EL-PFDD Meeting Host: Platelet Disorder Support Association | Meeting ReportExternal Link Disclaimer | July 26, 2019 |
Immune Thrombotic Thrombocytopenic Purpura (iTTP) | EL-PFDD Meeting Host: The Ree Wyn Foundation | Report pending | January 26, 2024 |
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 |
Juvenile Huntington’s Disease (JHD) | Patient Listening Session | Patient Listening Session Summary | February 10, 2023 |
Juvenile Idiopathic Arthritis (JIA) | EL-PFDD Meeting Host: The Arthritis Foundation and the Childhood Arthritis and Rheumatology Research Alliance (CARRA) | Meeting ReportExternal Link Disclaimer | April 2, 2018 |
KCNT1-Related Epilepsy | Patient Listening Session | Patient Listening Session Summary | April 22, 2024 |
Kennedy’s Disease (KD)/ Spinal and bulbar muscular atrophy (SBMA) | EL-PFDD Meeting Host: Kennedy’s Disease Association | Meeting ReportExternal Link Disclaimer | November 9, 2022 |
Kidney Xenotransplantation | EL-PFDD Meeting Host: National Kidney Foundation | Meeting ReportExternal Link Disclaimer | November 9, 2023 |
Krabbe Disease | EL-PFDD Meeting Host: National Organization for Rare Disorders (NORD) | Meeting ReportExternal Link Disclaimer | 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 |
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 ReportExternal Link Disclaimer | September 23, 2022 |
Long COVID | FDA-led PFDD Meeting | April 25, 2023 | |
Long QT Syndrome (LQTS) | EL-PFDD Meeting Host: SADS Foundation | Meeting ReportExternal Link Disclaimer | June 11, 2024 |
Low-Grade Serous Ovarian Cancer (LGSOC) | EL-PFDD Meeting Host: STAAR Ovarian Cancer Foundation | Meeting ReportExternal Link Disclaimer | October 13, 2023 |
Lung Cancer | FDA-led PFDD Meeting |
| June 28, 2013 |
Lupus | EL-PFDD Meeting Host: Lupus and Allied Diseases Association, Lupus Foundation of America, and the Lupus Research Alliance | Meeting ReportExternal Link Disclaimer | September 25, 2017 |
Lupus Nephritis (LN) | EL-PFDD Meeting Host: National Kidney Foundation | Report pending | October 11, 2024 |
Lupus – Diversity in Clinical Trials | Patient Listening Session | March 31, 2021 – Session 1 April 2, 2021 – Session 2 April 6, 2021 – Session 3 | |
Major Depressive Disorder (MDD) | EL-PFDD Meeting Host: Depression and Bipolar Support Alliance | Meeting ReportExternal Link Disclaimer | November 16, 2018 |
Mastocytosis | Patient Listening Session | Patient Listening Session Summary | September 28, 2021 |
Membranous Nephropathy (MN) | EL-PFDD Meeting Host: National Kidney Foundation and NephCure Kidney International | Meeting ReportExternal Link Disclaimer | August 27, 2021 |
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 ReportExternal Link Disclaimer | October 21, 2022 |
Mitochondrial Disease | EL-PFDD Meeting Host: United Mitochondrial Disease Foundation | Meeting ReportExternal Link Disclaimer | March 29, 2019 |
Myeloproliferative Neoplasms (MPNs) | EL-PFDD Meeting Host: MPN Research Foundation | Meeting ReportExternal Link Disclaimer | September 16, 2019 |
Myotonic Dystrophy (DM) | EL-PFDD Meeting Host: Myotonic Dystrophy Foundation | Meeting ReportExternal Link Disclaimer | September 15, 2016 |
Myotubular and Centronuclear Myopathy (MTM/CNM) | EL-PFDD Meeting Host: MTM-CNM Family Connection and Where There’s A Will There’s A Cure | MeetingExternal Link Disclaimer | August 3, 2021 |
Narcolepsy | FDA-led PFDD Meeting |
| September 24, 2013 |
Narcolepsy and Idiopathic Hypersomnia (IH) | Patient Listening Session | Patient Listening Session Summary | August 8, 2022 |
Neurofibromatosis (NF) | Patient Listening Session | Patient Listening Session Summary | June 13, 2019 |
Neurofibromatosis Type 1 (NF1)/ Cutaneous Neurofibroma (cNF) | Patient Listening Session | Patient Listening Session Summary | May 21, 2024 |
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 ReportExternal Link Disclaimer | March 18, 2019 |
Non-ketotic Hyperglycinemia (NKH) | Patient Listening Session | Patient Listening Session Summary | December 15, 2023 |
Non-Tuberculous Mycobacterial (NTM) Lung Infections | FDA-led PFDD Meeting |
| October 15, 2015 |
Nonalcoholic Steatohepatitis (NASH) | EL-PFDD Meeting Host: Global Liver Institute | Meeting ReportExternal Link Disclaimer | November 4, 2021 |
Obstructive Sleep Apnea (OSA) | EL-PFDD Meeting Host: American Sleep Apnea Association | Meeting ReportExternal Link Disclaimer | 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 below. 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 ReportExternal Link Disclaimer | 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 ReportExternal Link Disclaimer | April 6, 2018 |
Pancreatitis | EL-PFDD Meeting Host: National Pancreas Foundation and FCS Foundation | Meeting ReportExternal Link Disclaimer | 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 |
Pediatric Low-Grade Glioma (pLGG) | EL-PFDD Meeting Host: Pediatric Brain Tumor Foundation | Meeting ReportExternal Link Disclaimer | February 23, 2024 |
Pelizaeus Merzbacher Disease (PMD) | Patient Listening Session | Patient Listening Session Summary | August 22, 2023 |
Pemphigus and Pemphigoid | EL-PFDD Meeting Host: International Pemphigus and Pemphigoid Foundation | Meeting ReportExternal Link Disclaimer | January 25, 2023 |
Pemphigus and Pemphigoid | Patient Listening Session | Patient Listening Session Summary | February 8, 2021 |
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 |
Phelan-McDermid Syndrome (PMS) | EL-PFDD Meeting Host: CureSHANK | Meeting ReportExternal Link Disclaimer | November 8, 2022 |
Polycystic Ovary Syndrome (PCOS) | EL-PFDD Meeting Host: PCOS Challenge: The National Polycystic Ovary Syndrome Association | Report pending | November 3, 2023 |
Polyglutamine Spinocerebellar Ataxias (SCAs) and Dentatorubal-Pallidoluysian Atrophy (DRPLA) | EL-PFDD Meeting Host: National Ataxia Foundation and CureDRPLA | Meeting ReportExternal Link Disclaimer | September 25, 2020 |
Pompe Disease | EL-PFDD Meeting Host: Muscular Dystrophy Association | Meeting ReportExternal Link Disclaimer | July 13, 2020 |
Post-finasteride Syndrome (PFS) | Patient Listening Session | Patient Listening Session Summary | June 2, 2023 |
Post-Transplant Lymphoproliferative Disorder (PTLD) | EL-PFDD Meeting Host: National Organization for Rare Disorders (NORD) | Meeting ReportExternal Link Disclaimer | May 4, 2022 |
Pouchitis after a total colectomy and ideal pouch anal anastomosis (IPAA) surgery | Patient Listening Session | Patient Listening Session Summary | January 14, 2023 |
Prader-Willi Syndrome (PWS) | EL-PFDD Meeting Host: PWSA|USA | Meeting ReportExternal Link Disclaimer | June 23, 2023 |
Pre-Diagnosis Genetic Amyotrophic Lateral Sclerosis (ALS) and Frontotemporal Degeneration (FTD) | Patient Listening Session | Patient Listening Session Summary | January 12, 2023 |
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 |
Primary Biliary Cholangitis (PBC) | EL-PFDD Meeting Host: Global Liver Institute | Meeting ReportExternal Link Disclaimer | February 4, 2022 |
Primary Hyperoxaluria (PH) | EL-PFDD Meeting Host: The Oxalosis and Hyperoxaluria Foundation | Meeting ReportExternal Link Disclaimer | October 5, 2020 |
Primary Sclerosing Cholangitis (PSC) | EL-PFDD Meeting Host: PSC Partners Seeking a Cure | Meeting ReportExternal Link Disclaimer | October 23, 2020 |
Progressive Multifocal Leukoencephalopathy (PML) | Patient Listening Session | July 22, 2020 – Session 1 September 11, 2020 – Session 2 | |
Proteus Syndrome | Patient Listening Session | Patient Listening Session Summary | December 1, 2022 |
Psoriasis | FDA-led PFDD Meeting |
| March 17, 2016 |
Pulmonary Alveola Proteinosis (PAP) | Patient Listening Session | Patient Listening Session Summary | July 8, 2020 |
Pulmonary Arterial Hypertension (PAH) | FDA-led PFDD Meeting |
| May 13, 2014 |
Pulmonary Sarcoidosis | Patient Listening Session | Patient Listening Session Summary | April 21, 2022 |
Pyruvate Dehydrogenase Complex Deficiency (PDCD) | Patient Listening Session | Patient Listening Session Summary | September 8, 2023 |
Pyruvate Kinase Deficiency | EL-PFDD Meeting Host: National Organization for Rare Disorders (NORD) | Meeting ReportExternal Link Disclaimer | September 20, 2019 |
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 ReportExternal Link Disclaimer | May 16, 2024 |
Recurrent Clostridium difficile Infections (rCDI) | Patient Listening Session | Patient Listening Session Summary | March 10, 2023 |
Recurrent Respiratory Papillomatosis (RRP) | Patient Listening Session | Patient Listening Session Summary | October 27, 2022 |
Reducing Cardiac Late Effects in Pediatric Cancer Survivors | EL-PFDD Meeting Host: Children’s Cancer Cause | Meeting ReportExternal Link Disclaimer | September 15, 2022 |
Rett Syndrome | EL-PFDD Meeting Host: Rett Syndrome Research Trust (RSRT) | Meeting ReportExternal Link Disclaimer | March 11, 2022 |
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 |
Sarcoidosis | EL-PFDD Meeting Host: Foundation for Sarcoidosis Research | Report pending | October 28, 2024 |
Sarcopenia | FDA-led PFDD Meeting |
| April 6, 2017 |
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 ReportExternal Link Disclaimer | November 2, 2022 |
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 ReportExternal Link Disclaimer | May 25, 2021 |
Short Bowel Syndrome (SBS) | Patient Listening Session | Patient Listening Session Summary | July 19, 2022 |
Sickle Cell Disease | Patient Listening Session | Patient Listening Session Summary | May 5, 2023 |
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 Cord Injury (SCI) | Patient Listening Session | Patient Listening Session Summary | April 24, 2023 |
Spinal Muscular Atrophy (SMA) | Patient Listening Session | Patient Listening Session Summary | August 4, 2022 |
Spinal Muscular Atrophy (SMA) | EL-PFDD Meeting Host: Cure SMA | Meeting ReportExternal Link Disclaimer | April 18, 2017 |
Spinocerebellar Ataxia Type 3 (SCA3) | Patient Listening Session | Patient Listening Session Summary | September 22, 2023 |
Stargardt Disease (STGD1) | Patient Listening Session | Patient Listening Session Summary | June 24, 2022 |
Stimulant Use Disorder | FDA-led PFDD Meeting |
| October 6, 2020 |
STXBP1 | EL-PFDD Meeting Host: STXBP1 Foundation | Meeting ReportExternal Link Disclaimer | October 20, 2023 |
Succinic Semialdehyde Dehydrogenase Deficiency (SSADHD) | EL-PFDD Meeting Host: SSADH Association | Meeting ReportExternal Link Disclaimer | July 8, 2022 |
SYNGAP1 | EL-PFDD Meeting Host: SYNGAP1 Foundation | Meeting ReportExternal Link Disclaimer | November 19, 2020 |
Systemic Sclerosis | FDA-led PFDD Meeting |
| October 13, 2020 |
Thalassemia | EL-PFDD Meeting Host: Cooley’s Anemia Foundation | Report Unavailable | July 7, 2017 |
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 ReportExternal Link Disclaimer | 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 |
Wiskott-Aldrich Syndrome (WAS) and X-Linked Thrombocytopenia (XLT) | EL-PFDD Meeting Host: Wiskott-Aldrich Foundation | Meeting ReportExternal Link Disclaimer | February 3, 2023 |
X-linked Adrenoleukodystrophy (ALD) | EL-PFDD Meeting Host: ALD Connect | Meeting ReportExternal Link Disclaimer | July 22, 2022 |
X-linked Retinitis Pigmentosa (XLRP) | EL-PFDD Meeting Host: Fighting Blindness Foundation | Meeting ReportExternal Link Disclaimer | June 7, 2022 |
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 ReportExternal Link Disclaimer | August 19, 2021 |
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