Condition-Specific Meeting Reports and Other Information Related to Patients' Experience
Primary Biliary Cholangitis (PBC)
EL-PFDD Meeting
Host: Global Liver Institute
Report pending
February 4, 2022
Food Allergies
EL-PFDD Meeting
Host: Food Allergy Research and Education (FARE)
Report pending
September 9, 2021
Asthma
EL-PFDD Meeting
Host: Asthma and Allergy Foundation of America (AAFA)
Report pending
September 20, 2021
Myotubular and Centro Nuclear Myopathy (MTM/CNM)
EL-PFDD Meeting
Host: MTM-CNM Family Connection and Where There’s A Will There’s A Cure
Report pending
August 3, 2021
Rett Syndrome
EL-PFDD Meeting
Host: Rett Syndrome Research Trust (RSRT)
Report pending
March 11, 2022
Membranous Nephropathy
EL-PFDD Meeting
Host: National Kidney Foundation and NephCure Kidney International
Report pending
August 27, 2021
Cancer Cachexia
EL-PFDD Meeting
Host: Cancer Support Community
Report pending
November 2, 2021
Chronic inflammatory Demyelinating Polyneuropathy (CIDP)
EL-PFDD Meeting
Host: GBS|CIDP Foundation International
March 25, 2022
Thalassemia
EL-PFDD Meeting
Host: Cooley’s Anemia Foundation
Report pending
July 7, 2017
Juvenile Idiopathic Arthritis
EL-PFDD Meeting
Host: Arthritis Foundation and Childhood Arthritis and Rheumatology Research Alliance (CARRA)
Report pending
August 2, 2018
Cystic Fibrosis
EL-PFDD Meeting
Host: Systic Fibrosis Research Inc.
Report pending
October 29, 2018
CDKL5 Deficiency Disorder
EL-PFDD Meeting
Host: International Foundation for CDKL5 Research and the Loulou Foundation
Report pending
November 1, 2019
Developmental and Epileptic Encephalopathy
EL-PFDD Meeting
Host: Lennox-gastaut Syndrome
Report pending
November 1, 2019
Primary Hyperoxaluria
EL-PFDD Meeting
Host: The Oxalosis and Hyperoxaluria Foundation
October 5, 2020
SYNGAP1- Related Intellectual Disability Disorder
EL-PFDD Meeting
Host: Bridge the Gap-SYNGAP Education and Research Foundation
Report pending
November 19, 2020
Post-Transplant Lymphoproliferative Disease (PTLD)
EL-PFDD Meeting
Host: NORD
Report pending
May 4, 2022
Bronchiolitis Obliterans Syndrome (BOS)
EL-PFDD Meeting
Host: Lung Transplant Foundation with NORD
Report pending
June 22, 2022
Succinic Semialdehyde Dehydrogenase Deficiency (SSADHD)
EL-PFDD Meeting
Host: SSADH Association
Report pending
July 8, 2022
X-Linked Retinitis Pigmentosa
EL-PFDD Meeting
Host: Fighting Blindness Foundation
Report pending
June 7, 2022
Congenital Muscular Dystrophy
EL-PFDD Meeting
Host: Cure CMD
Report pending
July 1, 2022
Hermansky-Pudlak Syndrome
EL-PFDD Meeting
Host: Hermansky-Pudlak Syndrome Network
Report pending
June 10, 2022
X-Linked Adrenoleukodystrophy (ALD)
EL-PFDD Meeting
Host: ALD Connect
Report pending
July 22, 2022
Acromegaly
EL-PFDD Meeting
Host: Acromegaly Community, Inc.
January 21, 2021
Acute Porphyrias
EL-PFDD Meeting
Host: American Porphyria Foundation
March 1, 2017
Adrenomyeloneuropathy (AMN)
Patient Listening Session
May 7, 2021
Adult Dermatomyositis
Patient Listening Session
April 26, 2022
Adult Polyglucosan Body Disease
Patient Listening Session
October 28, 2021
Alopecia Areata
FDA-led PFDD Meeting
September 11, 2017
Alpha-1 Antitrypsin
FDA-led PFDD Meeting
September 29, 2015
Alport Syndrome
EL-PFDD Meeting
Host: National Kidney Foundation and the Alport Syndrome Foundation
August 3, 2018
Amyloidosis
EL-PFDD Meeting
Host: The Amyloidosis Research Consortium
November 16, 2015
Amyotrophic Lateral Sclerosis (ALS)
N/A
N/A
Autism
FDA-led PFDD Meeting
May 4, 2017
Barth Syndrome
EL-PFDD Meeting
Host: The Barth Syndrome Foundation
July 18, 2018
Biliary Atresia, Progressive Familial Intrahepatic Cholestasis, Wilson’s Disease
Patient Listening Session
November 5, 2018
Breast Cancer
FDA-led PFDD Meeting
April 2, 2015
Celiac Disease
Patient Listening Session
February 20, 2019
Cerebral Cavernous Malformation (CCM)
Patient Listening Session
November 6, 2019
Cerebrotendinous Xanthomatosis (CTX)
EL-PFDD Meeting
Host: United Leukodystrophy Foundation of America
September 14, 2021
Chagas Disease
FDA-led PFDD Meeting
April 28, 2015
Charcot-Marie-Tooth and Inherited Neuropathies
EL-PFDD Meeting
Host: Hereditary Neuropathy Foundation
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
September 13, 2018
Childhood Cerebral Adrenal Leukodystrophy (CCALD)
Patient Listening Session
November 13, 2019
Chronic Fatigue Syndrome/Myalgic Encephalomyelitis
FDA-led PFDD Meeting
April 25, 2013
Chronic Pain
FDA-led PFDD Meeting
July 9, 2018
Clear Cell Sarcoma
Patient Listening Session
Summary unavailable
September 10, 2020
Complement 3 Glomerulopathy (C3G)
EL-PFDD Meeting
Host: National Kidney Foundation
August 4, 2017
Cystic Fibrosis – nonsense mutations
Patient Listening Session
July 15, 2021
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
September 23, 2019
Dravet Syndrome
EL-PFDD Meeting
Host: The Dravet Syndrome Foundation
February 3, 2022
Fabry Disease
Patient Listening Session
December 4, 2018
Facioscapulohumeral Muscular Dystrophy (FSHD)
EL-PFDD Meeting
Host: FSHD Society
June 29, 2020
Female Sexual Dysfunction
FDA-led PFDD Meeting
October 27, 2014
Fibrodysplasia Ossificans Progressiva (FOP)
Patient Listening Session
May 29, 2019
Fibromyalgia
FDA-led PFDD Meeting
March 26, 2014
Focal Segmental Glomerulosclerosis
EL-PFDD Meeting
Host: National Kidney Foundation and NephCure Kidney International
August 28, 2020
Fragile X Syndrome
EL-PFDD Meeting
Host: National Fragile X Foundation
March 3, 2021
Friedreich’s Ataxia
EL-PFDD Meeting
Host: Friedreich’s Ataxia Research Alliance
June 2, 2017
Frontotemporal Degeneration (FTD)
EL-PFDD Meeting
Host: Association for Frontotemporal Degeneration (AFTD)
March 5, 2021
Functional Gastrointestinal Disorders
FDA-led PFDD Meeting
May 11, 2015
Gastroparesis
Patient Listening Session
December 2, 2019
Gene Therapy as a Treatment Modality for Hemophilia
Patient Listening Session
October 23, 2018
Glycogen Storage Disease (GSD) Type 1 – Adult Patients
Patient Listening Session
March 18, 2021
Glycogen Storage Disease (GSD) Type 1 – Caregivers to Pediatric Patients
Patient Listening Session
March 25, 2021
Glycogen Storage Disease Type 1B
Patient Listening Session
March 3, 2022
Gorlin Syndrome
Patient Listening Session
November 9, 2020
Gorlin Syndrome
EL-PFDD Meeting
Host: Gorlin Syndrome Alliance
October 8, 2021
Guillain-Barre’ Syndrome (GBS)
Patient Listening Session
September 29, 2020
Healthcare Challenges and Unmet Medical Needs of Transgender Adults
Patient Listening Session
June 1, 2021
Healthcare Challenges and Unmet Medical Needs of Transgender Adolescents
Patient Listening Session
June 29, 2021
Hemophilia A, B, and Other Heritable Bleeding Disorders
FDA-led PFDD Meeting
September 22, 2014
Hepatitis B
EL-PFDD Meeting
Host: Hepatitis B Foundation
June 9, 2020
Hereditary Angioedema
FDA-led PFDD Meeting
September 25, 2017
Homocystinuria (HCU)
Patient Listening Session
June 26, 2020
Human Immunodeficiency Virus (HIV)
FDA-led PFDD Meeting
June 14, 2013
Hunter Syndrome
Patient Listening Session
February 4, 2020
Hunter Syndrome (MPS II)
N/A
N/A
Huntington’s Disease
FDA-led PFDD Meeting
September 22, 2015
Hypereosinophilic Syndromes
EL-PFDD Meeting
Host: American Partnership for Eosinophilic Disorders
March 23, 2018
Hyperhidrosis
EL-PFDD Meeting
Host: International Hyperhidrosis Society
November 13, 2017
Hypertrophic Cardiomyopathy
EL-PFDD Meeting
Host: Hypertrophic Cardiomyopathy Association
June 26, 2020
Hypomyelination with Atrophy of Basal Ganglia and Cerebellum (H-ABC)
Patient Listening Session
May 9, 2022
Hypophosphatemia
EL-PFDD Meeting
Host: XLH Network
October 5, 2018
Hypothalamic Obesity
Patient Listening Session
October 22, 2021
Ichthyosis
Patient Listening Session
September 17, 2021
Idiopathic Pulmonary Fibrosis
FDA-led PFDD Meeting
September 26, 2014
IgA Nephropathy
EL-PFDD Meeting
Host: National Kidney Foundation and the IgA Nephropathy Foundation of America
August 19, 2019
Immune Thrombocytopenia
EL-PFDD Meeting
Host: Platelet Disorder Support Association
July 26, 2019
Inborn Errors of Metabolism
FDA-led PFDD Meeting
June 10, 2014
Inclusion Body Myositis (IBM)
Patient Listening Session
March 5, 2020
Juvenile Dermatomyositis (JDM)
Patient Listening Session
Summary unavailable
March 16, 2021
Krabbe Disease
EL-PFDD Meeting
Host: National Organization for Rare Disorders (NORD)
October 29, 2020
Late Onset GM2 Gangliosidoses (Tay-Sachs and Sandhoff Diseases)
Patient Listening Session
January 15, 2021
Limb Girdle Muscular Dystrophies (LGMD)
Patient Listening Session
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
September 25, 2017
Major Depressive Disorder
EL-PFDD Meeting
Host: Depression and Bipolar Support Alliance
November 16, 2018
Mastocytosis
Patient Listening Session
September 28, 2021
Mitochondrial Disease
EL-PFDD Meeting
Host: United Mitochondrial Disease Foundation
March 29, 2019
Myeloproliferative Neoplasms
EL-PFDD Meeting
Host: MPN Research Foundation
September 16, 2019
Myotonic Dystrophy
EL-PFDD Meeting
Host: Myotonic Dystrophy Foundation
September 15, 2016
Narcolepsy
FDA-led PFDD Meeting
September 24, 2013
Neurofibromatosis (NF)
Patient Listening Session
June 13, 2019
Neuronopathic Gaucher Disease (Types 2 & 3)
Patient Listening Session
September 9, 2021
Neuropathic Pain Associated with Peripheral Neuropathy
FDA-led PFDD Meeting
June 10, 2016
Niemann-Pick Type C
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
March 18, 2019
Nonalcoholic Steatohepatitis
EL-PFDD Meeting
Host: Global Liver Institute
November 4, 2021
Non-tuberculous Mycobacterial Lung Infections
FDA-led PFDD Meeting
October 15, 2015
Obstructive Sleep Apnea
EL-PFDD Meeting
Host: American Sleep Apnea Association
June 8, 2018
Ocular Melanoma
Patient Listening Session
January 27, 2020
Opioid Use Disorder
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
Patient Listening Session
August 7, 2019
Osteoarthritis
EL-PFDD Meeting
Host: Arthritis Foundation
March 8, 2017
Osteogenesis Imperfecta (OI)
Patient Listening Session
September 17, 2019
Pachyonychia Congenita
EL-PFDD Meeting
Host: Pachyonychia Congenita Project
April 6, 2018
Pancreatitis
EL-PFDD Meeting
Host: National Pancreas Foundation and FCS Foundation
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
February 8, 2021
Polyglutamine Spinocerebellar Ataxias (SCAs) and Dentatorubal-Pallidoluysian Atrophy (DRPLA)
EL-PFDD Meeting
Host: National Ataxia Foundation and CureDRPLA
September 25, 2020
Pompe Disease
EL-PFDD Meeting
Host: Muscular Dystrophy Association
July 13, 2020
Primary Ciliary Dyskinesia
Patient Listening Session
Summary coming soon
September 1, 2021
Primary Sclerosing Cholangitis
EL-PFDD Meeting
Host: PSC Partners Seeking a Cure
October 23, 2020
Progressive Multifocal Leukoencephalopathy
Patient Listening Session
July 22, 2020 – Session 1
September 11, 2020 – Session 2
Psoriasis
FDA-led PFDD Meeting
March 17, 2016
Pulmonary Alveolar Proteinosis
Patient Listening Session
Summary unavailable
July 8, 2020
Pulmonary Arterial Hypertension
FDA-led PFDD Meeting
May 13, 2014
Pulmonary Sarcoidosis
Patient Listening Session
April 21, 2022
Pyruvate Kinase Deficiency
EL-PFDD Meeting
Host: National Organization for Rare Disorders (NORD)
September 20, 2019
Sanfilippo Syndrome
Patient Listening Session
May 13, 2019
Sanfilippo Syndrome – Pediatric
Patient Listening Session
October 17, 2019
Sarcopenia
FDA-led PFDD Meeting
April 6, 2017
SCN2A Related Disorders
Patient Listening Session
April 28, 2021
Sensorineural Hearing Loss
EL-PFDD Meeting
Host: Hearing Loss Association of America
May 25, 2021
Sickle Cell Disease
FDA-led PFDD Meeting
February 7, 2014
Smith-Magenis Syndrome
Patient Listening Session
August 12, 2020
Spinal Muscular Atrophy
EL-PFDD Meeting
Host: Cure SMA
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 (TK2)
Patient Listening Session
January 31, 2022
Tuberous Sclerosis Complex
EL-PFDD Meeting
Host: Tuberous Sclerosis Alliance
June 21, 2017
Valosin Containing Protein Disease (VCP)
Patient Listening Session
May 5, 2020
Vascular Ehlers-Danlos Syndrome (VEDS)
Patient Listening Session
October 7, 2020
Vitiligo
FDA-led PFDD Meeting
March 8, 2021
Von Hippel Lindau (VHL)
Patient Listening Session
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
August 19, 2021
X-Linked Myotubular Myopathy (XLMTM)
Patient Listening Session
Summary unavailable
August 3, 2020
