Limb Girdle Muscular Dystrophy 2E/R4: SRP-9003 Gene Therapy

We are studying a new gene therapy to see if it improves muscle function in people with Limb Girdle Muscular Dystrophy 2E/R4. The trial will evaluate its safety and effectiveness over time.

>2 years Confirmation phase (III)No Placebo Cost Reimbursement Partially Remote Investigational Medicines Oral InjectionNeurologyOrthopedics and Traumatology

What diseases are being studied?

Health conditions and diseases that the clinical trial is designed to study and treat.

Who can participate in the clinical trial?

These questions help us understand your situation so we can let the trial team know whether you might be a potential match. This is not a medical evaluation and is not part of the official screening, the study doctor will make the final decision..

What medicines are being studied?

Medicines that are officially approved for use in the European Union and considered safe by EMA. These medicines are already on the market and may be tested for new conditions or populations.

Prednisone Eg

Prednisone Eg is a steroid medicine that reduces inflammation and treats allergic, autoimmune, and other inflammatory conditions.

What active substances are being studied?

Active substances that are officially approved for use in the European Union and considered safe by EMA. These substances are already on the market and may be tested for new conditions or populations.

Prednisone

Prednisone is a steroid substance that reduces inflammation and suppresses the immune system for conditions like allergies, asthma, and autoimmune disorders.

What experimental substances are being tested?

Experimental substances that are being investigated in this clinical trial. These are not yet approved for general use.

SrP-9003

Bidridistrogene Xeboparvovec

What similar medicines might you know?

Don't worry if you don't know the medicines tested in the trial. There is a chance you have heard about the similar medicines. Check the list below to see if you are familiar with any of them.

Brucorten

Cison

Cortancyl

Where is the clinical trial being conducted?

Locations and medical facilities where this clinical trial is currently recruiting or conducting research. Select the locations where you would like to participate.

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UZ Leuven

Neurology

Heverlee, Belgium

Universitair Ziekenhuis Gent

Neuromusculair referentiecentrum

Ledeberg, Belgium

Universitaetsklinikum Essen AöR

Klinik für Kinderheilkunde I, Sozialpädiatrisches Zentrum (SPZ)

Essen, Germany

Sponsor: Sarepta Therapeutics Inc.

Last updated: Nov 26, 2025

Disclaimer: Parts of this content have been automatically extracted from the EU Clinical Trials registry. While we strive for accuracy, please always contact the trial site or sponsor directly for correct and official information before making any decisions about participation. View on EU Clinical Trials.