Discover various promising treatments in development to address the unmet needs of people with Charcot–Marie–Tooth disease.

This novel treatment approach has resulted in compelling preclinical and clinical data generalized myasthenia gravis, spinal muscular atrophy, Charcot-Marie Tooth disease, sarcopenia and holds ...

The US biopharma will discontinue development of the Exondys 51 (eteplirsen) follow up treatment to focus on other treatment ...

Others, including those diagnosed with Duchenne muscular dystrophy (DMD), typically have lived into their teens and 20s. Muscular dystrophy, a genetic disease that affects the muscles, can lead to ...

muscular dystrophy）、三好氏遠端肌肉無力症／遠端型肌肉失養症（英语：Distal muscular dystrophy）和艾梅氏肌失養症（英语：Emery-Dreifuss muscular dystrophy））被划分为肌营养不良症。 符合肌營養不良症的體徵和症狀是： 漸進性肌肉萎縮 平衡感不佳 染色体倒位 sulfatase ...

Although often considered to be diseases of childhood, adult forms of muscular dystrophy occur and represent a spectrum of disease. Muscular dystrophies are a genetically heterogeneous group of ...

The power to influence or even direct people’s behavior or the course of events is part of a typical definition of “control.” When living with a chronic illness, as I am with limb-girdle muscular ...

As a child, Michael Rudnicki liked to dismantle clocks. He would take them apart, piece by piece, to see what made them tick. That curiosity has been a driving force throughout the celebrated ...

Novartis had one eye on the struggles its peers have encountered in the muscular dystrophy space when it ... neuromuscular disease and neurodegeneration. “We are now, I think, at a place where ...