There is a simple discount patient access scheme for vamorolone. NHS organisations can get details on the Commercial Access and Pricing (CAP) Portal. Non-NHS organisations can contact ...

Muscular dystrophy is diagnosed using several tests. These may include: Muscle biopsy. A small piece of muscle tissue is removed and examined to confirm the diagnosis or rule out another muscle ...

What Is Duchenne Muscular Dystrophy? Muscular dystrophies are a ... your child might have other symptoms, including: The muscle problems can cause cramps at times, but in general, DMD isn ...

A gene-editing therapy designed to correct a defect in the DYSF gene — one that’s associated with a form of limb-girdle muscular dystrophy, or LGMD — was found to restore dysferlin protein levels and ...

Researchers at the Experimental and Clinical Research Center (ECRC), a joint institution of the Max Delbrück Center and Charité – Universitätsmedizin Berlin, have developed a promising gene-editing ...

Facioscapulohumeral muscular dystrophy (FSHD) is an autosomal dominant muscle disease with a prevalence of 1/10,000–1/20,000 (Lunt and Harper, 1991; Deenen et al., 2014; Tihaya et al., 2023). It is ...

People with certain mutations in the gene coding for dysferlin develop muscular dystrophy—a group of muscle wasting diseases that affect thousands around the world. Professor Simone Spuler and ...

People with certain mutations in the gene coding for dysferlin develop muscular dystrophy – a group of muscle wasting diseases that affect thousands around the world. Professor Simone Spuler and ...