Amish nemaline myopathy

Last reviewed

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ORPHA:98902OMIM:605355G71.2
Who is this for?
Show terms as
1Active trials1Specialists8Treatment centers

Where are you in your journey?

UniteRare data is sourced from FDA.gov, ClinicalTrials.gov, Orphanet, OMIM, and NORD.
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Overview

Clinical phenotype terms— hover any for plain English:

Shoulder flexion contractureHP:0003044Hip contractureHP:0003273Progressive muscle weaknessHP:0003323Type 1 muscle fiber predominanceHP:0003803Proximal amyotrophyHP:0007126
Orphanet ↗OMIM ↗NORD ↗

FDA & Trial Timeline

1 event
Sep 2018WiTNNess - TNNT1 Myopathy Natural History Study

Clinic for Special Children

TrialRECRUITING

Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.

Treatments

No FDA-approved treatments are currently listed for Amish nemaline myopathy.

1 clinical trialare actively recruiting — trials can provide access to cutting-edge therapies.

View clinical trials →

Clinical Trials

1 recruitingView all trials with filters →
Other1 trial
WiTNNess - TNNT1 Myopathy Natural History Study
Actively Recruiting
PI: Kevin Strauss, MD (Clinic for Special Children) · Sites: Gordonville, Pennsylvania

Specialists

1 foundView all specialists →
KM
Kevin Strauss, MD
GORDONVILLE, PA
Specialist
PI on 1 active trial

Treatment Centers

8 centers
🏥 NORD

Baylor College of Medicine Rare Disease Center

Baylor College of Medicine

📍 Houston, TX

🏥 NORD

Stanford Medicine Rare Disease Center

Stanford Medicine

📍 Stanford, CA

🔬 UDN

NIH Clinical Center Undiagnosed Diseases Program

National Institutes of Health

📍 Bethesda, MD

🔬 UDN

UCLA UDN Clinical Site

UCLA Health

📍 Los Angeles, CA

🔬 UDN

Baylor College of Medicine UDN Clinical Site

Baylor College of Medicine

📍 Houston, TX

🔬 UDN

Harvard/MGH UDN Clinical Site

Massachusetts General Hospital

📍 Boston, MA

🏥 NORD

Mayo Clinic Center for Individualized Medicine

Mayo Clinic

📍 Rochester, MN

👤 Mayo Clinic Center for Individualized Medicine

🏥 NORD

UCLA Rare Disease Day Program

UCLA Health

📍 Los Angeles, CA

Travel Grants

No travel grants are currently matched to Amish nemaline myopathy.

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Community

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Latest news about Amish nemaline myopathy

Disease timeline:

New recruiting trial: WiTNNess - TNNT1 Myopathy Natural History Study

A new clinical trial is recruiting patients for Amish nemaline 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 Amish nemaline myopathy

Are there clinical trials for Amish nemaline myopathy?

Yes — 1 recruiting clinical trial is currently listed for Amish nemaline myopathy on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.

Which specialists treat Amish nemaline myopathy?

1 specialists and care centers treating Amish nemaline myopathy are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.