The latest developments in muscular dystrophy research

SUNFISH study on spinal muscular atrophy

Roche is involved in a two-part SUNFISH study of RG7916, in people with type 2 or 3 spinal muscular atrophy. The initial part has been completed, which showed RG7916 was safe. The second part will determine if the dose, chosen in the first stage, is safe and effective.

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Update on Acceleron studies

Acceleron Pharma is working on two drugs ACE-083 and ACE-2494 which would increase muscle mass. ACE-083 is in phase 2 trials and is used on people suffering from facioscapulohumeral dystrophy and Charcot-Marie-Tooth disease. Preclinical research on ACE-2494 has given a positive outcome.

AMO trial shows positive results on myotonic dystrophy

The phase 2 trial by AMO has shown that patients with congenital and juvenile onset myotonic dystrophy who received tideglusib (AMO-02) improved clinically.

Update on myasthenia gravis

Alexion Pharmaceuticals’ phase 3 study on eculizumab (Regain) on patients with refractory generalised myasthenia gravis has shown a good outcome so far.

CRISPR and congenital muscular dystrophy

A clinical study showed CRISPR can correct the genetic defect in mice with merosin-deficient congenital muscular dystrophy resolving all paralysis. This has been published in the journal, Nature Medicine.

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Advancements in Duchenne muscular dystrophy

Capricor Therapeutics has made progress with its phase 1 and 2 clinical trial, HOPE. Participants with Duchenne muscular dystrophy who received CAP-1002 had improvement in cardiovascular and upper limb function.There is a plan to initiate HOPE-2, a double-blind, placebo-controlled clinical trial in 2018.

Patients who took edasalonexent during the phase 2 trial (MoveDMD) by Catabasis Pharmaceuticals showed a reduced progression of the disease after 36 weeks. An initial study failed to yield results when participants took the drug for 12 weeks. There are steps to initiate phase 3 clinical trials after discussions with the Food and Drug Administration (FDA), which is the regulatory body.

Muscle biopsies on patients treated with golodirsen showed increased dystrophin production. This was during the early phase clinical trials performed by Sarepta Therapeutics. Companies such as can help with early phase trials including adaptive phase 1 clinical studies.

Upcoming clinical trials

Audentes Therapeutics will start a phase 1/2 trial for boys with X-linked myotubular myopathy to determine if AT132, gene therapy is effective.

MitoBridge today will initiate a trial to find out if the drug MA-0211 can treat Duchenne muscular dystrophy.

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