Company Releases Early Data and Plans to Boost Dose in Duchenne Muscular Dystrophy Trial

According to a story from pm360online.com, the life sciences company Solid Biosciences has released some preliminary findings from its Phase 1/2 clinical trial of SGT-001. This investigational product is in development as a treatment for Duchenne muscular dystrophy. Solid Biosciences is committed to the development of new treatments for Duchenne muscular dystrophy.

About Duchenne Muscular Dystrophy

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, boys are mostly affected, with girls 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.

Preliminary Data

The company has always planned to increase dose as the trial continued and is currently working to begin the process promptly. The preliminary data that Solid has released comes from the first three participants in the trial that were dosed so far. These patients were biopsied after a three month period. In these patients, microdystrophin was detected, but at very low levels. 

Dystrophin deficiency is the root mechanism that causes symptoms of Duchenne muscular dystrophy. This early data suggests that these patients could benefit from receiving a larger dose of SGT-001. This experimental treatment aims to compensate for a patient’s lack of dystrophin by delivering a synthetic transgene variant called microdystrophin, which can take the place of dystrophin. This could allow SGT-001 to slow or possible halt the progression of Duchenne muscular dystrophy regardless of the extent of progression or the mutation that is present.


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