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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 Other information includes proposed draft guidance relating to patient experience data, natural history studies, or other condition-specific background on condition and discussion of unmet medical need.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 MeetingResource(s)Meeting Date
Non-ketotic Hyperglycinemia (NKH)Patient Listening SessionPatient Listening Session SummaryDecember 15, 2023
Adult-onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP)EL-PFDD Meeting
Host: Sisters’ Hope Foundation
Patient Listening Session SummaryJanuary 17, 2024
SarcoidosisEL-PFDD Meeting
Host: Foundation for Sarcoidosis Research
Report pendingOctober 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 pendingNovember 13, 2024
Dementia with Lewy Bodies (DLB)EL-PFDD Meeting
Host: Lewy Body Dementia Association
Report pendingOctober 15, 2024
Lupus Nephritis (LN)EL-PFDD Meeting
Host: National Kidney Foundation
Report pendingOctober 11, 2024
Classic Congenital Adrenal Hyperplasia (CAH)EL-PFDD Meeting
Host: CARES Foundation
Report pendingOctober 1, 2024
ThalassemiaEL-PFDD Meeting
Host: Cooley’s Anemia Foundation
Report UnavailableJuly 7, 2017
Neurofibromatosis Type 1 (NF1)/ Cutaneous Neurofibroma (cNF)Patient Listening SessionPatient Listening Session SummaryMay 21, 2024
KCNT1-Related EpilepsyPatient Listening SessionPatient Listening Session SummaryApril 22, 2024
Attention-Deficit/Hyperactivity Disorder (ADHD)Patient Listening SessionPatient Listening Session SummaryMarch 15, 22, and 29, 2024
Long QT Syndrome (LQTS)EL-PFDD Meeting
Host: SADS Foundation
Meeting ReportJune 11, 2024
Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT)EL-PFDD Meeting
Host: SADS Foundation
Meeting ReportJune 11, 2024
Adult Dermatomyositis (DM)EL-PFDD Meeting
Host: Myositis Support and Understanding
Report pendingJune 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 pendingMay 28, 2024
Rare, Autoimmune Peripheral Neuropathies: Guillain-Barre Syndrome (GBS), Multifocal Motor Neuropathy (MMN), and Anti-myeline-associated glycoprotein (Anti-MAG) neuropathyEL-PFDD Meeting
Host: GBS|CIDP Foundation International
Meeting ReportMay 16, 2024
Idiopathic Hypersomnia (IH)EL-PFDD Meeting
Host: Sleep Consortium
Meeting ReportApril 11, 2024
Congenital Cytomegalovirus Infection (cCMV)Patient Listening SessionPatient Listening Session SummaryApril 4, 2024
Pelizaeus Merzbacher Disease (PMD)Patient Listening SessionPatient Listening Session SummaryAugust 22, 2023
HypoparathyroidismEL-PFDD Meeting
Host: HypoPARAthyroidism Association
Meeting ReportMarch 5, 2024
Dry Age-Related Macular Degeneration (Dry AMD)EL-PFDD Meeting
Host: Foundation Fighting Blindness
Report pendingMarch 1, 2024
Pediatric Low-Grade Glioma (pLGG)EL-PFDD Meeting
Host: Pediatric Brain Tumor Foundation
Meeting ReportFebruary 23, 2024
GM2 gangliosidoses (Tay-Sachs and Sandhoff diseases)EL-PFDD Meeting
Host: National Tay-Sachs & Allied Diseases Association (NTSAD)
Meeting ReportFebruary 15, 2024
Immune Thrombotic Thrombocytopenic Purpura (iTTP)EL-PFDD Meeting
Host: The Ree Wyn Foundation
Report pendingJanuary 26, 2024
Pyruvate Dehydrogenase Complex Deficiency (PDCD)Patient Listening SessionPatient Listening Session SummarySeptember 8, 2023
Atypical Hemolytic Uremic Syndrome (aHUS)Patient Listening SessionPatient Listening Session SummarySeptember 21, 2023
Epidermolysis Bullosa (EB)EL-PFDD Meeting
Host: Dystrophic Epidermolysis Bullosa Research Association of America (Debra of America)
MeetingApril 6, 2018
Spinal Cord Injury (SCI)Patient Listening SessionPatient Listening Session SummaryApril 24, 2023
C. difficile InfectionsPatient Listening SessionPatient Listening Session SummaryMarch 10, 2023
Kidney XenotransplantationEL-PFDD Meeting
Host: National Kidney Foundation
Meeting ReportNovember 9, 2023
Polycystic Ovary Syndrome (PCOS)EL-PFDD Meeting
Host: PCOS Challenge: The National Polycystic Ovary Syndrome Association
Report pendingNovember 3, 2023
Hypermobile Ehlers-Danlos Syndrome (hEDS) and Hypermobility spectrum disorders (HSD)EL-PFDD Meeting
Host: The Ehlers-Danlos Society
Report pendingOctober 31, 2023
Classical Homocystinuria (HCU)EL-PFDD Meeting
Host: HCU Network America
Report pendingOctober 27, 2023
STXBP1EL-PFDD Meeting
Host: STXBP1 Foundation
Meeting ReportOctober 20, 2023
Low-Grade Serous Ovarian Cancer (LGSOC)EL-PFDD Meeting
Host: STAAR Ovarian Cancer Foundation
Meeting ReportOctober 13, 2023
Cystic Fibrosis (CF)EL-PFDD Meeting
Host: Cystic Fibrosis Research Institute
Meeting ReportOctober 29, 2018
Spinocerebellar Ataxia Type 3 (SCA3)Patient Listening SessionPatient Listening Session SummarySeptember 22, 2023
Developmental and Epileptic Encephalopathy (DEE)EL-PFDD Meeting
Host: Lennox-Gastaut Syndrome Foundation
MeetingNovember 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 ReportNovember 1, 2019
Autosomal Recessive Polycystic Kidney Disease (ARPKD)EL-PFDD Meeting
Host: PKD Foundation
Meeting ReportAugust 29, 2023
Carcinoid SyndromePatient Listening SessionPatient Listening Session SummaryApril 27, 2023
Canavan DiseasePatient Listening SessionPatient Listening Session SummaryMarch 21, 2023
Post-finasteride Syndrome (PFS)Patient Listening SessionPatient Listening Session SummaryJune 2, 2023
Prader-Willi Syndrome (PWS)EL-PFDD Meeting
Host: PWSA|USA
Meeting ReportJune 23, 2023
Arrhythmogenic Right Ventricular Cardiomyopathy/Dysplasia (ARVC/D)EL-PFDD Meeting
Host: SADS Foundation
Meeting ReportJune 20, 2023
Recurrent Clostridium difficile Infections (rCDI)Patient Listening SessionPatient Listening Session SummaryMarch 10, 2023
Sickle Cell DiseasePatient Listening SessionPatient Listening Session SummaryMay 5, 2023
Pulmonary Alveola Proteinosis (PAP)Patient Listening SessionPatient Listening Session SummaryJuly 8, 2020
Bronchopulmonary Dysplasia (BPD)Patient Listening SessionPatient Listening Session SummaryMarch 20, 2023
Juvenile Idiopathic Arthritis (JIA)

EL-PFDD Meeting

Host: The Arthritis Foundation and the Childhood Arthritis and Rheumatology Research Alliance (CARRA)

Meeting ReportApril 2, 2018
Long COVIDFDA-led PFDD MeetingApril 25, 2023
Congenital Disorders of Glycosylation (CDG)Patient Listening SessionPatient Listening Session SummaryJanuary 30, 2023
Juvenile Huntington’s Disease (JHD)Patient Listening SessionPatient Listening Session SummaryFebruary 10, 2023
Pouchitis after a total colectomy and ideal pouch anal anastomosis (IPAA) surgeryPatient Listening SessionPatient Listening Session SummaryJanuary 14, 2023
SYNGAP1

EL-PFDD Meeting

Host: SYNGAP1 Foundation

Meeting ReportNovember 19, 2020
Pre-Diagnosis Genetic Amyotrophic Lateral Sclerosis (ALS) and Frontotemporal Degeneration (FTD)Patient Listening SessionPatient Listening Session SummaryJanuary 12, 2023
Proteus SyndromePatient Listening SessionPatient Listening Session SummaryDecember 1, 2022
Stargardt Disease (STGD1)Patient Listening SessionPatient Listening Session SummaryJune 24, 2022
Recurrent Respiratory Papillomatosis (RRP)Patient Listening SessionPatient Listening Session SummaryOctober 27, 2022
Wiskott-Aldrich Syndrome (WAS) and X-Linked Thrombocytopenia (XLT)EL-PFDD Meeting
Host: Wiskott-Aldrich Foundation
Meeting ReportFebruary 3, 2023
Autoimmune Hepatitis (AIH) EL-PFDD Meeting
Host: Autoimmune Hepatitis Association
MeetingJanuary 27, 2023
Pemphigus and PemphigoidEL-PFDD Meeting
Host: International Pemphigus and Pemphigoid Foundation
Meeting ReportJanuary 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 ReportJanuary 24, 2023
Narcolepsy and Idiopathic Hypersomnia (IH)Patient Listening SessionPatient Listening Session SummaryAugust 8, 2022
Hypophosphatasia (HPP)EL-PFDD Meeting
Host: Soft Bones, Inc., The US
Hypophosphatasia Foundation
Meeting ReportNovember 15, 2022
GM1 gangliosidosisEL-PFDD Meeting
Host: Cure GM1 Foundation
Meeting ReportOctober 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 ReportOctober 21, 2022
SchizophreniaEL-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 ReportNovember 2, 2022
Phelan-McDermid Syndrome (PMS)EL-PFDD Meeting
Host: CureSHANK
Meeting ReportNovember 8, 2022
Kennedy’s Disease (KD)/ Spinal and bulbar muscular atrophy (SBMA)EL-PFDD Meeting
Host: Kennedy’s Disease Association
Meeting ReportNovember 9, 2022
Short Bowel Syndrome (SBS)Patient Listening SessionPatient Listening Session SummaryJuly 19, 2022
Huntington’s Disease (HD) Pre-Symptomatic PopulationPatient Listening SessionPatient Listening Session SummaryJuly 25, 2022
Spinal Muscular Atrophy (SMA)Patient Listening SessionPatient Listening Session SummaryAugust 4, 2022
Alstrom Syndrome (AS)

EL-PFDD Meeting

Host: Alstrom International

Meeting ReportSeptember 22, 2022
Reducing Cardiac Late Effects in Pediatric Cancer Survivors

EL-PFDD Meeting

Host: Children’s Cancer Cause

Meeting ReportSeptember 15, 2022
Fabry Disease

EL-PFDD Meeting

Host: National Kidney Foundation and Fabry Support & Information Group

Report pendingSeptember 19, 2022
Galactosemia

EL-PFDD Meeting

Host: National Organization for Rare Disorders (NORD)

Meeting ReportSeptember 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 ReportSeptember 23, 2022
Primary Biliary Cholangitis (PBC)EL-PFDD Meeting
Host: Global Liver Institute
Meeting ReportFebruary 4, 2022
Food AllergiesEL-PFDD Meeting
Host: Food Allergy Research and Education (FARE)
Meeting ReportSeptember 9, 2021
AsthmaEL-PFDD Meeting
Host: Asthma and Allergy Foundation of America (AAFA)
Meeting ReportSeptember 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
MeetingAugust 3, 2021
Rett SyndromeEL-PFDD Meeting
Host: Rett Syndrome Research Trust (RSRT)
Meeting ReportMarch 11, 2022
Membranous Nephropathy (MN)EL-PFDD Meeting
Host: National Kidney Foundation and NephCure Kidney International
Meeting ReportAugust 27, 2021
Cancer CachexiaEL-PFDD Meeting
Host: Cancer Support Community
Meeting ReportNovember 2, 2021
Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)EL-PFDD Meeting
Host: GBS|CIDP Foundation International
Meeting ReportMarch 25, 2022
Primary Hyperoxaluria (PH)EL-PFDD Meeting
Host: The Oxalosis and Hyperoxaluria Foundation
Meeting ReportOctober 5, 2020
Post-Transplant Lymphoproliferative Disorder (PTLD)EL-PFDD Meeting
Host: National Organization for Rare Disorders (NORD)
Meeting ReportMay 4, 2022
Bronchiolitis Obliterans Syndrome (BOS)EL-PFDD Meeting
Host: Lung Transplant Foundation
Meeting ReportJune 22, 2022
Succinic Semialdehyde Dehydrogenase Deficiency (SSADHD)EL-PFDD Meeting
Host: SSADH Association
Meeting ReportJuly 8, 2022
X-linked Retinitis Pigmentosa (XLRP)EL-PFDD Meeting
Host: Fighting Blindness Foundation
Meeting ReportJune 7, 2022
Congenital Muscular Dystrophy (CMD)EL-PFDD Meeting
Host: Cure CMD
Report pendingJuly 1, 2022
Hermansky-Pudlak Syndrome (HPS)EL-PFDD Meeting
Host: Hermansky-Pudlak Syndrome Network
Meeting ReportJune 10, 2022
X-linked Adrenoleukodystrophy (ALD)EL-PFDD Meeting
Host: ALD Connect
Meeting ReportJuly 22, 2022

Acromegaly

 

EL-PFDD Meeting
Host: Acromegaly Community, Inc.
Meeting ReportJanuary 21, 2021
Acute PorphyriasEL-PFDD Meeting
Host: American Porphyria Foundation
Meeting ReportMarch 1, 2017
Adrenomyeloneuropathy (AMN)Patient Listening SessionPatient Listening Session SummaryMay 7, 2021
Adult Dermatomyositis (DM)Patient Listening SessionPatient Listening Session SummaryApril 26, 2022
Adult Polyglucosan Body Disease (APBD)Patient Listening SessionPatient Listening Session SummaryOctober 28, 2021
Alopecia AreataFDA-led PFDD MeetingSeptember 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 ReportAugust 3, 2018
AmyloidosisEL-PFDD Meeting
Host: The Amyloidosis Research Consortium
Meeting ReportNovember 16, 2015
Amyotrophic Lateral Sclerosis (ALS)N/AIMPACT ALS Survey ReportN/A
Autism (ASD)FDA-led PFDD Meeting

 

May 4, 2017
Barth Syndrome (BTHS)EL-PFDD Meeting
Host: The Barth Syndrome Foundation
Meeting ReportJuly 18, 2018
Breast CancerFDA-led PFDD Meeting

 

April 2, 2015
Celiac DiseasePatient Listening SessionPatient Listening Session SummaryFebruary 20, 2019
Cerebral Cavernous Malformation (CCM)Patient Listening SessionPatient Listening Session SummaryNovember 6, 2019
Cerebrotendinous Xanthomatosis (CTX)EL-PFDD Meeting
Host: United Leukodystrophy Foundation of America
Meeting ReportSeptember 14, 2021
Chagas DiseaseFDA-led PFDD Meeting

 

April 28, 2015
Charcot-Marie-Tooth and Inherited Neuropathies (CMT/IN)EL-PFDD Meeting
Host: Hereditary Neuropathy Foundation
Meeting ReportSeptember 28, 2018
Chemotherapy-Induced Hearing LossEL-PFDD Meeting
Host: Children’s Cause for Cancer Advocacy, Children’s Brain Tumor Foundation, Mattie Miracle Cancer Foundation, and Momcology
Meeting ReportSeptember 13, 2018
Childhood Cerebral Adrenal Leukodystrophy (CCALD)Patient Listening SessionPatient Listening Session SummaryNovember 13, 2019
Chronic Fatigue Syndrome/Myalgic Encephalomyelitis (CFS & ME)FDA-led PFDD Meeting

 

April 25, 2013
Chronic PainFDA-led PFDD Meeting

 

July 9, 2018
Complement 3 Glomerulopathy (C3G)EL-PFDD Meeting
Host: National Kidney Foundation
Meeting ReportAugust 4, 2017
Cystic Fibrosis (CF) – nonsense mutationsPatient Listening SessionPatient Listening Session SummaryJuly 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 ReportSeptember 23, 2019
Dravet Syndrome (DS)EL-PFDD Meeting
Host: The Dravet Syndrome Foundation
Meeting ReportFebruary 3, 2022
Fabry DiseasePatient Listening SessionPatient Listening Session SummaryDecember 4, 2018
Facioscapulohumeral Muscular Dystrophy (FSHD)EL-PFDD Meeting
Host: FSHD Society
Meeting ReportJune 29, 2020
Female Sexual Dysfunction (FSD)FDA-led PFDD Meeting

 

October 27, 2014
Fibrodysplasia Ossificans Progressiva (FOP)Patient Listening SessionPatient Listening Session SummaryMay 29, 2019
FibromyalgiaFDA-led PFDD Meeting

 

March 26, 2014
Focal Segmental Glomerulosclerosis (FSGS)EL-PFDD Meeting
Host: National Kidney Foundation and NephCure Kidney International
Meeting ReportAugust 28, 2020
Fragile X Syndrome (FXS)EL-PFDD Meeting
Host: National Fragile X Foundation
Meeting ReportMarch 3, 2021
Friedreich’s Ataxia (FA)EL-PFDD Meeting
Host: Friedreich’s Ataxia Research Alliance
Meeting ReportJune 2, 2017
Frontotemporal Degeneration (FTD)EL-PFDD Meeting
Host: Association for Frontotemporal Degeneration (AFTD)
Meeting ReportMarch 5, 2021
Functional Gastrointestinal (GI) DisordersFDA-led PFDD Meeting

 

May 11, 2015
GastroparesisPatient Listening SessionPatient Listening Session SummaryDecember 2, 2019
Gene Therapy as a Treatment Modality for HemophiliaPatient Listening SessionPatient Listening Session SummaryOctober 23, 2018
Glycogen Storage Disease (GSD) Type 1 – Adult PatientsPatient Listening SessionPatient Listening Session SummaryMarch 18, 2021
Glycogen Storage Disease (GSD) Type 1 – Caregivers to Pediatric PatientsPatient Listening SessionPatient Listening Session SummaryMarch 25, 2021
Glycogen Storage Disease (GSD) Type 1BPatient Listening SessionPatient Listening Session SummaryMarch 3, 2022
Gorlin Syndrome (GS)Patient Listening SessionPatient Listening Session SummaryNovember 9, 2020
Gorlin Syndrome (GS)EL-PFDD Meeting
Host: Gorlin Syndrome Alliance
Meeting ReportOctober 8, 2021
Guillain-Barre’ Syndrome (GBS)Patient Listening SessionPatient Listening Session SummarySeptember 29, 2020
Healthcare Challenges and Unmet Medical Needs of Transgender AdultsPatient Listening SessionPatient Listening Session SummaryJune 1, 2021
Healthcare Challenges and Unmet Medical Needs of Transgender AdolescentsPatient Listening SessionPatient Listening Session SummaryJune 29, 2021
Hemophilia A, Hemophilia B, von Willebrand Disease (VWB), and Other Heritable Bleeding DisordersFDA-led PFDD Meeting

 

September 22, 2014
Chronic Hepatitis BEL-PFDD Meeting
Host: Hepatitis B Foundation
Meeting ReportJune 9, 2020
Hereditary Angioedema (HAE)FDA-led PFDD Meeting

 

September 25, 2017
Homocystinuria (HCU)Patient Listening SessionPatient Listening Session SummaryJune 26, 2020
Human Immunodeficiency Virus (HIV)FDA-led PFDD MeetingJune 14, 2013
Hunter Syndrome (MPS II)Patient Listening SessionPatient Listening Session SummaryFebruary 4, 2020
Hunter Syndrome (MPS II)N/AToileting Ability Survey ReportN/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 ReportMarch 23, 2018
Hyperhidrosis (Hh)EL-PFDD Meeting
Host: International Hyperhidrosis Society
Meeting ReportNovember 13, 2017
Hypertrophic Cardiomyopathy (HCM)EL-PFDD Meeting
Host: Hypertrophic Cardiomyopathy Association
Meeting ReportJune 26, 2020
Hypomyelination with Atrophy of Basal Ganglia and Cerebellum (H-ABC)Patient Listening SessionPatient Listening Session SummaryMay 9, 2022
HypophosphatemiaEL-PFDD Meeting
Host: XLH Network
Meeting ReportOctober 5, 2018
Hypothalamic Obesity (HO)Patient Listening SessionPatient Listening Session SummaryOctober 22, 2021
IchthyosisPatient Listening SessionPatient Listening Session SummarySeptember 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 ReportAugust 19, 2019
Immune Thrombocytopenia (ITP)EL-PFDD Meeting
Host: Platelet Disorder Support Association
Meeting ReportJuly 26, 2019
Inborn Errors of Metabolism (IEM)FDA-led PFDD Meeting

 

June 10, 2014
Inclusion Body Myositis (IBM)Patient Listening SessionPatient Listening Session SummaryMarch 5, 2020
Krabbe DiseaseEL-PFDD Meeting
Host: National Organization for Rare Disorders (NORD)
Meeting ReportOctober 29, 2020
Late Onset GM2 gangliosidoses (Tay-Sachs and Sandhoff Diseases)Patient Listening SessionPatient Listening Session SummaryJanuary 15, 2021
Limb-Girdle Muscular Dystrophies (LGMD)Patient Listening SessionPatient Listening Session SummaryOctober 20, 2020
Lung CancerFDA-led PFDD Meeting

 

June 28, 2013
Lupus – Diversity in Clinical TrialsPatient Listening Session

March 31, 2021 – Session 1

April 2, 2021 – Session 2

April 6, 2021 – Session 3

LupusEL-PFDD Meeting
Host: Lupus and Allied Diseases Association, Lupus Foundation of America, and the Lupus Research Alliance
Meeting ReportSeptember 25, 2017
Major Depressive Disorder (MDD)EL-PFDD Meeting
Host: Depression and Bipolar Support Alliance
Meeting ReportNovember 16, 2018
MastocytosisPatient Listening SessionPatient Listening Session SummarySeptember 28, 2021
Mitochondrial DiseaseEL-PFDD Meeting
Host: United Mitochondrial Disease Foundation
Meeting ReportMarch 29, 2019
Myeloproliferative Neoplasms (MPNs)EL-PFDD Meeting
Host: MPN Research Foundation
Meeting ReportSeptember 16, 2019
Myotonic Dystrophy (DM)EL-PFDD Meeting
Host: Myotonic Dystrophy Foundation
Meeting ReportSeptember 15, 2016
NarcolepsyFDA-led PFDD Meeting

 

September 24, 2013
Neurofibromatosis (NF)Patient Listening SessionPatient Listening Session SummaryJune 13, 2019
Neuronopathic Gaucher Disease (nGD) (Types 2 & 3)Patient Listening SessionPatient Listening Session SummarySeptember 9, 2021
Neuropathic Pain Associated with Peripheral NeuropathyFDA-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 ReportMarch 18, 2019
Nonalcoholic Steatohepatitis (NASH)EL-PFDD Meeting
Host: Global Liver Institute
Meeting ReportNovember 4, 2021
Non-Tuberculous Mycobacterial (NTM) Lung InfectionsFDA-led PFDD Meeting

 

October 15, 2015
Obstructive Sleep Apnea (OSA)EL-PFDD Meeting
Host: American Sleep Apnea Association
Meeting ReportJune 8, 2018
Ocular Melanoma (OM)Patient Listening SessionPatient Listening Session SummaryJanuary 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 SessionPatient Listening Session SummaryAugust 7, 2019
Osteoarthritis (OA)EL-PFDD Meeting
Host: Arthritis Foundation
Meeting ReportMarch 8, 2017
Osteogenesis Imperfecta (OI)Patient Listening SessionPatient Listening Session SummarySeptember 17, 2019
Pachyonychia Congenita (PC)EL-PFDD Meeting
Host: Pachyonychia Congenita Project
Meeting ReportApril 6, 2018
PancreatitisEL-PFDD Meeting
Host: National Pancreas Foundation and FCS Foundation
Meeting ReportMarch 3, 2020
Parkinson’s DiseaseFDA-led PFDD Meeting

 

September 22, 2015
Patients Who Have Received an Organ TransplantFDA-led PFDD Meeting

 

September 27, 2016
Pemphigus and PemphigoidPatient Listening SessionPatient Listening Session SummaryFebruary 8, 2021
Polyglutamine Spinocerebellar Ataxias (SCAs) and Dentatorubal-Pallidoluysian Atrophy (DRPLA)EL-PFDD Meeting
Host: National Ataxia Foundation and CureDRPLA
Meeting ReportSeptember 25, 2020
Pompe DiseaseEL-PFDD Meeting
Host: Muscular Dystrophy Association
Meeting ReportJuly 13, 2020
Primary Sclerosing Cholangitis (PSC)EL-PFDD Meeting
Host: PSC Partners Seeking a Cure
Meeting ReportOctober 23, 2020
Progressive Multifocal Leukoencephalopathy (PML)Patient Listening Session

July 22, 2020 – Session 1

September 11, 2020 – Session 2

PsoriasisFDA-led PFDD Meeting

 

March 17, 2016
Pulmonary Arterial Hypertension (PAH)FDA-led PFDD Meeting

 

May 13, 2014
Pulmonary SarcoidosisPatient Listening SessionPatient Listening Session SummaryApril 21, 2022
Pyruvate Kinase DeficiencyEL-PFDD Meeting
Host: National Organization for Rare Disorders (NORD)
Meeting ReportSeptember 20, 2019
Sanfilippo SyndromePatient Listening SessionPatient Listening Session SummaryMay 13, 2019
Sanfilippo Syndrome – PediatricPatient Listening SessionPatient Listening Session SummaryOctober 17, 2019
SarcopeniaFDA-led PFDD Meeting

 

April 6, 2017
SCN2A Related DisordersPatient Listening SessionPatient Listening Session SummaryApril 28, 2021
Sensorineural Hearing Loss (SNHL)EL-PFDD Meeting
Host: Hearing Loss Association of America
Meeting ReportMay 25, 2021
Sickle Cell DiseaseFDA-led PFDD Meeting

 

February 7, 2014
Smith-Magenis Syndrome (SMS)Patient Listening SessionPatient Listening Session SummaryAugust 12, 2020
Spinal Muscular Atrophy (SMA)EL-PFDD Meeting
Host: Cure SMA
Meeting ReportApril 18, 2017
Stimulant Use DisorderFDA-led PFDD Meeting

 

October 6, 2020
Systemic SclerosisFDA-led PFDD Meeting

 

October 13, 2020
Thymidine Kinase 2 Deficiency (TK2)Patient Listening SessionPatient Listening Session SummaryJanuary 31, 2022
Tuberous Sclerosis Complex (TSC)EL-PFDD Meeting
Host: Tuberous Sclerosis Alliance
Meeting ReportJune 21, 2017
Valosin Containing Protein Disease (VCP)Patient Listening SessionPatient Listening Session SummaryMay 5, 2020
Vascular Ehlers-Danlos Syndrome (VEDS)Patient Listening SessionPatient Listening Session SummaryOctober 7, 2020
VitiligoFDA-led PFDD Meeting

 

March 8, 2021
Von Hippel Lindau (VHL)Patient Listening SessionPatient Listening Session SummaryJune 11, 2020
XerostomiaEL-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 ReportAugust 19, 2021

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