Weekly Spotlight - 14.11.24

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In the News

Sarepta Halts Duchenne Drug Amidst Rising Gene Therapy Success

Sarepta Therapeutics has halted development of its Duchenne drug SRP-5051 due to safety concerns and the success of its gene therapy, Elevidys. Elevidys sales rose significantly after FDA approval expansion, promising a bright future for Duchenne treatment and offering hope to affected families.

Solid Biosciences Expands Duchenne Trial, Enhancing Hope for Young Patients

Solid Biosciences has expanded the INSPIRE DUCHENNE trial, now including 43 participants aged 4 to under 12. The evaluation period extends to 18 months, enhancing assessment of SGT-003's impact. Three new sites are added, with initial data expected in early 2025. A webinar is scheduled for November.

Join the PPMD Adult Advisory Committee for Duchenne and Becker Support

The PPMD Adult Advisory Committee seeks adults with Duchenne or Becker muscular dystrophy to join their mission. Members, aged 18 and over, will engage in advocacy, mentorship, and education. Applications, including a resume, are due by 25th November. Participation involves bi-weekly Zoom meetings and occasional in-person events.

Muscular Dystrophy Association Celebrates Family Caregivers' Vital Role in November

The Muscular Dystrophy Association highlights National Family Caregivers Awareness Month, emphasising caregivers' vital role in supporting those with neuromuscular diseases. Through resources and personal stories, like those of the Eby and Lopez families, MDA offers practical and emotional support, fostering a community of care and understanding.