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Latest treatment for neuromuscular diseases available at CHoR

Sunday, January 29, 2017

Amy D. Harper, MDThe FDA has recently approved two drugs for rare neuromuscular diseases.

Exondys 51 (Etperlirsen) became the first drug given approval by the FDA for Duchenne muscular dystrophy in September 2016. The pathway for the use of this weekly intravenous drug is currently in progress. Children's Hospital of Richmond at VCU is working to identify patients who may be candidates for this therapy, with hopes of having the first patient receive the new drug within the next two months.

Not all boys with Duchenne muscular dystrophy are candidates for this treatment as it is gene-specific, however, there are other candidate genes and therapies on the horizon. Several new trials for Duchenne muscular dystrophy will be starting at CHoR in the spring.

Additionally, Spinraza (nusinersen) was FDA approved in December 2016 for the treatment of all forms of spinal muscular atrophy. It is an intrathecal medication which requires careful administration and monitoring. Similar to Exondys, steps are being taken at CHoR to develop a pathway for the administration of this medication. This process involves coordination among pediatric specialties throughout CHoR.

Although there are no current cures for Duchenne muscular dystrophy or spinal muscular atrophy, these new FDA-approved drugs are providing hope for the future!

Schedule an appointment with CHoR neuromuscular specialist Dr. Amy Harper to learn more about these new treatments: 804-828-CHOR (2467)

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