We are pleased to announce that there is an open spot for a virtual TACT (TREAT-NMD Advisory Committee for Therapeutics) review on Tuesday, July 29th, 2025. This is a unique opportunity […]
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Highlighting Cure HSPB8: Advocacy for a Rare Disease
HSPB8 Myopathy, also known as Myofibrillar Myopathy Type 13 with Rimmed Vacuoles (MFM13, OMIM: #621078) is an ultrarare, autosomal dominant muscle-wasting disease that typically begins in early adulthood. Despite its [...]
SMA White Paper updates to support SMA newborn screening
In SMA, newborn screening (NBS), dramatically change the life of young babies. SMA Europe has created an Alliance involving clinicians, patients, pharmaceutical industry and DNA testing producers, to promote SMA […]
2025 Annual Curators Meeting (ACM)
The 2025 Annual Curators Meeting (ACM) took place in Milan from February 10th to 12th, a fitting tribute to outgoing chair Anna Ambrosini, who calls the city home. This year’s […]
TREAT-NMD End of Year Newsletter 2024
With deepest gratitude, we extend our thanks to the incredible individuals who have generously shared their expertise, guidance, and precious time, driving forward our mission to transform the diagnosis, care, and treatment of those living with neuromuscular diseases. TREAT-NMD’s work would not be possible without the incredible contributions of our global community—thank you for being a part of this journey.
Developing Care Guidelines for DMD Patients Following an Early Diagnosis
Duchenne muscular dystrophy (DMD) is the most prevalent form of muscular dystrophy, affecting approximately 1 in 5,000 boys, resulting in progressive muscle weakness which significantly impacts the lifespan of those […]
