Overview
FDA & Trial Timeline
5 eventsUniversity of Nevada, Las Vegas — NA
University of South Carolina — NA
Second Affiliated Hospital, School of Medicine, Zhejiang University — NA
Elpida Therapeutics SPC — PHASE1, PHASE2
Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.
Treatments
No FDA-approved treatments are currently listed for Congenital myopathy.
View clinical trials →Clinical Trials
View all trials with filters →No actively recruiting trials found for Congenital myopathy at this time.
New trials open frequently. Follow this disease to get notified.
Rare Disease Specialist
Rare Disease Specialist
Treatment Centers
8 centersBaylor College of Medicine Rare Disease Center ↗
Baylor College of Medicine
📍 Houston, TX
🏥 NORDStanford Medicine Rare Disease Center ↗
Stanford Medicine
📍 Stanford, CA
🔬 UDNNIH Clinical Center Undiagnosed Diseases Program ↗
National Institutes of Health
📍 Bethesda, MD
🔬 UDNUCLA UDN Clinical Site ↗
UCLA Health
📍 Los Angeles, CA
🔬 UDNBaylor College of Medicine UDN Clinical Site ↗
Baylor College of Medicine
📍 Houston, TX
🔬 UDNHarvard/MGH UDN Clinical Site ↗
Massachusetts General Hospital
📍 Boston, MA
🏥 NORDMayo Clinic Center for Individualized Medicine ↗
Mayo Clinic
📍 Rochester, MN
👤 Mayo Clinic Center for Individualized Medicine
🏥 NORDUCLA Rare Disease Day Program ↗
UCLA Health
📍 Los Angeles, CA
Travel Grants
No travel grants are currently matched to Congenital myopathy.
Community
No community posts yet. Be the first to share your experience with Congenital myopathy.
Start the conversation →Latest news about Congenital myopathy
Disease timeline:
New recruiting trial: Effect of Physical Training in Individuals With Hypokalemic and Hyperkalemic Periodic Paralysis
A new clinical trial is recruiting patients for Congenital myopathy
New recruiting trial: Dose Response of Exercise for Arthritis Management
A new clinical trial is recruiting patients for Congenital myopathy
New recruiting trial: Natural History Study - Mitochondrial Disease
A new clinical trial is recruiting patients for Congenital myopathy
New recruiting trial: Melpida: Recombinant Adeno-associated Virus (serotype 9) Encoding a Codon Optimized Human AP4M1 Transgene (hAP4M1opt)
A new clinical trial is recruiting patients for Congenital myopathy
Caregiver Resources
NORD Caregiver Resources
Support, advocacy, and financial assistance for caregivers of rare disease patients.
Mental Health Support
Rare disease caregiving can be isolating. Connect with counseling and peer support.
Family & Caregiver Grants
Financial assistance programs specifically for caregivers of rare disease patients.
Social Security Disability
Learn how rare disease patients may qualify for SSDI/SSI benefits.
Common questions about Congenital myopathy
Which specialists treat Congenital myopathy?
22 specialists and care centers treating Congenital myopathy are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.