According to a story from the CureDuchenne Blog, CureDuchenne founder and CEO Debra Miller announced that the organization has recently entered into a new partnership with the biotechnology company Myosana Therapeutics. CureDuchenne is officially investing in the company’s ongoing research. Myosana aims to develop treatments for Duchenne muscular dystrophy and other neuromuscular disorders. The company is aiming to develop a proprietary method of treatment that offers greater efficacy.
About Duchenne Muscular Dystrophy (DMD)
Duchenne muscular dystrophy is a neuromuscular disease, and it is one of the more severe types of muscular dystrophy. It is characterized by progressive muscle weakness that usually begins around age four and worsens quickly. As an X-linked genetic disease, males are mostly affected, with females only occasionally displaying mild symptoms. The disease is caused by mutations of the dystrophin gene. Symptoms of Duchenne muscular dystrophy include falling, abnormal walking posture, eventual loss of walking ability, muscle fiber deformities, intellectual disability (not in all cases), enlargement of the tongue and calf muscles, skeletal deformities, muscle atrophy, heart abnormalities, and difficulty with breathing. Treatment includes a variety of medications and therapies that can help alleviate symptoms and slow disease progression. Lifespan is usually into the thirties with good care. Better treatments for this disease are urgently needed. To learn more about Duchenne muscular dystrophy, click here.
Myosana aims to the develop a treatment that prioritizes cardiac and skeletal muscle and intends to deliver full-length dystrophin in order to maintain muscle strength. This represents an advancement over current treatments, which often used a shortened dystrophin gene, as the full size gene is very long and can’t be delivered with viral vectors easily. Myosana also aims to develop a treatment that will work for all Duchenne muscular dystrophy patients, not just those with a specific mutation.
While the company is still in the early stages of researching the development of this new treatment, it has the potential to be a serious game changer for treating this disorder if it winds up completing development successfully. To learn more about the goals of the company in developing this therapy, click here.