Weekly Spotlight - 09.01.25

In the news

Italfarmaco's Duvyzat approved to significantly aid Duchenne battle in UK

The MHRA has approved Italfarmaco's Duvyzat for treating Duchenne muscular dystrophy in the UK. It's for patients who start treatment while still ambulatory and conditionally for non-ambulatory patients. Positive results from the EPIDYS trial support its approval, aiming to quickly make it available in the UK.

Innovative Hope as Molecular Patch Development Aims to Transform Muscular Dystrophy

A new molecular patch aims to treat muscular dystrophy by skipping harmful changes to the COL6A1 gene, which damages muscle cells. This early-stage research hopes to deliver effective treatment and potentially slow the condition's progression, offering a promising new future for muscle wasting conditions.

Running Together for 20 Years as We Celebrate PPMD’s Impactful Race Programme

PPMD's Race to End Duchenne Programme celebrates its 20th year. With 185 participants in the 2025 Walt Disney World Marathon, it's raised over £18 million. This effort has extended lives, developed therapies, and provided vital support to families.

Pioneering Gene Therapy Offers New Hope for Muscular Dystrophy Patients

Gene therapy shows promise for those with muscular dystrophy, offering hope for muscle repair. Using CRISPR, researchers restored protein function in mice, getting ready for human trials. Though not a full cure, this method targets specific muscles, aiming to heal and enhance patient mobility.