Sveikata

Accelerated Approval of Elevidys (SRP-9001) for Children with DMD

Peticija adresuota
Europäische Arzneimittel-Agentur (EMA)

37 552 parašai

Rinkimas baigtas

37 552 parašai

Rinkimas baigtas

  1. Pradėta birželio 2023
  2. Rinkimas baigtas
  3. Parengti pateikimą
  4. Dialogas su gavėju
  5. Sprendimas

Peticija adresuojama: Europäische Arzneimittel-Agentur (EMA)

Lady's and Gentlemen
As the undersigned of this petition, we would like to speak out in favor of Elevidys (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.

Priežastis

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

Dėkojame už palaikymą, Kai Andre-Lüchtefeld iš Bad Laer
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Informacija apie peticiją

Peticija prasidėjo: 2023-06-30
Peticija baigiasi: 2024-09-18
Regione: Europos Sąjunga
tema: Sveikata

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  • Support for the gene therapy petition

    Dear Sir or Madam

    I hope this email reaches you in good health and good spirits. I am writing to you on behalf of our petition group, which is campaigning for rapid approval of Sarepta Therapeutics SRP-9001 (Elevidys) gene therapy in Germany.

    As you may know, this gene therapy was approved in the USA almost six months ago. Since then, we have received more and more positive feedback from various publicly accessible forums supporting patients with Duchenne muscular dystrophy (DMD) about the effect of the gene therapy.

    Some parents report that after 8 weeks of gene therapy administration, there are no more signs of the "Growers' sign", which is typical for boys with DMD and describes the difficulty... toliau

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Zdravilo!! za uspešno zdravljene naših otrok!

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