Company Continues to Back an Experimental Therapy for Duchenne Muscular Dystrophy Despite Clinical Hold

According to a story from, the pharmaceutical company Solid Biosciences is pledging to continue developing its investigation drug candidate SGT-001 for Duchenne muscular dystrophy. This is despite its clinical trials getting off to a dangerous start; the first patient given a dose of the treatment was rushed to the hospital after experiencing a a drop in first platelet count and then red blood cell count as well.
Duchenne muscular dystrophy (DMD), while generally rare, is the most widespread variant of muscular dystrophy. Muscular dystrophy is characterized by progressively worsening muscle degeneration due to a lack of dystrophin in the muscles. This is a disease that caused by a genetic mutation. Wasting generally occurs first in the upper legs and pelvis, and later the upper arms. Boys are most commonly affected and generally patients lose their ability to walk by age 12. Scoliosis is also common in this condition. Life expectancy is significantly affected, with most patients living into their mid-20s. With top of the line care, patients occasionally survive into the 30s or 40s. Treatment focuses on slowing disease progression, but there is no cure. To learn more about Duchenne muscular dystrophy, click here.

After the hospitalization of the first patient, the incident was categorized as a Suspected Unexpected Serious Adverse Reaction (SUSAR). The U.S. Food and Drug Administration has put the trial on a clinical hold as a result, which, at this juncture, has halted any continuation of the study. The company is hoping to continue working the development of SGT-001, and will be working closely with the FDA to understand the necessary requirements to get the trial progressing again.

The news also resulted in plunging stocks for the company. The dramatic plummet has resulted in lawsuits for Solid Biosciences as well. The company has also acknowledged that getting the trial back on track might be only part of the solution to getting the development of the new therapy back to smooth sailing. One of the company advisers also resigned due to the fallout from the incident.

New treatments for DMD are desperately needed, so hopefully SGT-001 will eventually get developed into a safe and effective treatment for this debilitating condition.

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