On Nov. 19, Santhera Pharmaceuticals released a letter to the Duchenne muscular dystrophy (DMD) community announcing its decision to suspend enrollment of new participants in the BreatheDMD Expanded Access Program (EAP) until the SIDEROS clinical trial is fully enrolled. The full letter follows. SIDEROS clinical trial and BreatheDMD program update: Dear Duchenne community, In February 2018, Santhera . . .
Genetic testing has a critical role in patients’ care as genetic information helps physicians to distinguish between types of neuromuscular disorders, informs prognosis and treatment decisions, and determines patients’ eligibility for new and emerging therapies. Selecting the right genetic test and incorporating genetic information to reach a diagnosis and formulate a treatment plan require deep . . .
On Nov. 10, Genentech, a member of the Roche Group, announced positive results from the second part of its pivotal phase 2/3 SUNFISH clinical trial assessing risdiplam in patients with spinal muscular atrophy (SMA) types 2 or 3. The study met its primary endpoint, which was change from baseline in the Motor Function Measure 32 (MFM-32) . . .
Today, Solid Biosciences released a letter to the Duchenne muscular dystrophy (DMD) community regarding the decision by the US Food and Drug Administration (FDA) to place the company’s IGNITE DMD clinical trial on hold because the latest child dosed experienced a serious adverse event (SAE). The full letter follows. Letter to the Duchenne Community: Update . . .
Today, Roche Genentech released a letter to the Duchenne muscular dystrophy (DMD) community regarding its decision to discontinue its DMD clinical development program. The full letter follows. Letter to the DMD community from Roche Genentech: Dear members of the Duchenne community, We are writing to share with you unfortunate news about our clinical development program . . .
At more than 150 of the leading healthcare institutions across the US, MDA Care Centers offer top clinical care to individuals living with muscular dystrophy, ALS, and other neuromuscular diseases. Providing access to this expert multidisciplinary care, however, is just one way that Care Centers serve the neuromuscular community. MDA Care Centers are conducting clinical . . .
On Oct. 30, AveXis released a letter to the spinal muscular atrophy (SMA) community regarding a decision by the US Food and Drug Administration (FDA) to place a partial hold on clinical trials for intrathecal (IT) administration of AVXS-101. The full letter follows. The order stops enrollment of patients in the high-dose group of AveXis’ . . .
Today marks the first day of open enrollment for the national Health Insurance Marketplace and many state health insurance marketplaces. If you purchase your health insurance from either the state or federal marketplace, it’s important that you take a look at the plans available for 2020 to ensure that they meet the needs of you . . .
Several biotech and pharma companies presented interim results at the 24th Annual Congress of the World Muscle Society (WMS) held earlier this month in Copenhagen, Denmark. This roundup covers a few of these updates. Catabasis Pharmaceuticals presented positive results from its phase 1/2 MoveDMD trial and open-label extension with edasalonexent in boys with Duchenne muscular . . .
Texas-based Reata Pharmaceuticals announced positive results of its pivotal phase 2 clinical trial (MOXIe) of omaveloxolone in patients with Friedreich’s ataxia (FA). Patients treated with omaveloxolone showed a statistically significant improvement in the modified Friedreich’s Ataxia Rating Scale mFARS (a scale used to measure neurological function) after 48 weeks of treatment compared to placebo, and . . .