Health

Accelerated Approval of SRP-9001 for Children with DMD

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Europäische Arzneimittel-Agentur (EMA)
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  1. Launched 30/06/2023
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Lady's and Gentlemen

As the undersigned of this petition, we would like to speak out in favor of SRP-9001 from Sarepta Therapeutics / Roche being approved quickly in Europe for children with Duchenne muscular dystrophy (DMD) who are able to walk. We believe this approval is urgently needed because every day counts for patients and the fastest possible treatment can mean a significant improvement in the quality of life for patients with DMD.

Reason

DMD is an inherited disease that can lead to a gradual deterioration in muscle function and ultimately to respiratory and cardiac impairment. There is currently no cure for DMD, and existing therapies are primarily aimed at relieving the symptoms of the disease. However, SRP-9001 offers a promising avenue to target the disease.

In the most common form, Duchenne muscular dystrophy, the prognosis is serious. Those affected usually die at the age of 20 to 25 from the progressive deterioration of the heart and respiratory muscles.

SRP-9001 has just been approved in the US and has shown significant improvements in patients with DMD in multiple studies. The rapid approval of SRP-9001 in Europe could give many European patients the same opportunity for an improved quality of life.

Therefore, we urge you to advocate for rapid approval of Sarepta Therapeutics' SRP-9001 in Europe for Duchenne muscular dystrophy. Every day matters for the patients with DMD and we believe that SRP-9001 represents a very good treatment option.

More information about SRP-9001

Thank you for your support, Kai Andre-Lüchtefeld from Bad Laer
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