Beta-sarcoglycan-related limb-girdle muscular dystrophy R4 Advocacy Hub | UniteRare

At a Glance

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Regulatory Watchboard

2 events

Jan 2024A Trial to Learn More About an Experimental Gene Therapy Called Bidridistrogene Xeboparvovec (SRP-9003) as a Possible Treatment for Limb Girdle Muscular Dystrophy 2E/R4

Sarepta Therapeutics, Inc. — PHASE3

TrialACTIVE NOT RECRUITING

Dec 2022A Gene Transfer Single Dose Study to Evaluate the Safety, Tolerability and Efficacy of SRP-9003 in Non-Ambulatory and Ambulatory Participants With Limb Girdle Muscular Dystrophy, Type 2E/R4 (Beta-Sarcoglycan [β-SG] Deficiency)

Sarepta Therapeutics, Inc. — PHASE1

TrialACTIVE NOT RECRUITING

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

Financial Assistance

FINANCIAL LANDSCAPE SUMMARY

Total programs

No financial assistance programs currently listed for Beta-sarcoglycan-related limb-girdle muscular dystrophy R4.
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Clinical Trial Landscape

2 active trials

1Phase 3

1Phase 1

2Total recruiting

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Recent News & Research

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Specialist Network

Top 3 by expertise

Nicholas Johnson, MD

Virginia Commonwealth University

📍 Richmond, Virginia

Score 75 trials

Jerry R Mendell, M.D., MD

Nationwide Children's Hospital

📍 COLUMBUS, OH

Score 32 trials

Linda Lowes, PT PhD

Nationwide Children's Hospital

Score 21 trial

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