CureDuchenne is investing $1 million in Entos Pharmaceuticals to support the development of a new DMD gene therapy.

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 ...

Entrada has secured the authorisation within the EU Clinical Trial Regulation to begin the Phase I/II trial of ENTR-601-45.

Months after causing safety concerns that led the FDA to hit a related trial with a clinical hold, a phase 2 trial of ...

An abnormality in the DMD gene causes Duchenne muscular dystrophy. The DMD gene produces dystrophin, a protein that maintains the structure of muscle cells. People with DMD have mutations (changes ...

Local intramuscular administration of the antisense oligonucleotide PRO051 in patients with Duchenne's muscular dystrophy with relevant mutations was previously reported to induce the skipping of ...

DMD treatment SAT-3247 was safe and well-tolerated, and showed signs of increasing muscle strength in five adults, Satellos ...

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, shared the following ...

A new gene therapy for Duchenne muscular dystrophy (DMD ... could be applied to other genetic diseases that are caused by mutations in large genes. The research was published in the journal ...

Figure 3: DNA sequence electrophoregrams representing the two mutations in the telethonin gene associated with LGMD 2G. Figure 5: Analysis of the telethonin protein in muscle samples. We performed ...