Gene Therapy: Dramatically improvement on children with myotubular myopathy

The gene therapy developed for children with myotubular myopathy has had astonishing results in clinical trials. Nine children have received the treatment until now and their neuromuscular function has improved dramatically. Most children can now sit on their own, four are now breathing without ventilators and three are making stepping movements. Myotubular Myopathy (MTM) is … Read more…

Another approval for clinical trials for centronuclear myopathies

Today, the Medicines and Healthcare products Regulatory Agency (MHRA) has approved clinical trials of the drug DYN101. This drug regulates the production of the enzyme dynamin 2 in the body. This could lead to an improvement in the symptoms of people with various centronuclear myopathies (CNM) (those with mutations in the genes MTM1 and DNM2). … Read more…