Neuromuscular

The professor at the University of Pittsburgh was awarded a $190,000 grant for mouse model research that specifically targets neuromuscular weakness in spinal muscular atrophy.

Take 5 minutes to catch up on NeurologyLive's highlights from the week ending April 17, 2020.

The chief medical advisor for the Muscular Dystrophy Association discussed how the ongoing novel coronavirus pandemic will have a lasting impact on the use of telemedicine in the neuromuscular community­­.

Neurology News Network for the week ending April 11, 2020.

Take 5 minutes to catch up on NeurologyLive's highlights from the week ending April 10, 2020.

The director of Experimental Neurotherapeutics, St. Jude Children's Research Hospital, Memphis, Tennessee, and principal investigator in the DEVOTE study discussed details of the trial and its potential impact for patients with spinal muscular atrophy.

The chief medical advisor for the Muscular Dystrophy Association detailed his experience with the process of having to manage patients and conduct research amid the COVID-19 pandemic.

Disease-modifying therapies used to treat several immune-mediated disorders in neurology may make patients more susceptible to the novel coronavirus.

The chief medical advisor for the Muscular Dystrophy Association spoke about the process of keeping patients updated on new information and how the MDA has taken steps to ensure the neuromuscular community gets the correct information.

The FDA has moved the PDUFA date for risdiplam back to August 24, 2020, as the agency works to review additional data that was made available from part 2 of the pivotal SUNFISH trial.

A pre-clinical study showed that supplementing diet with amino acid successfully staves off signs of amyotrophic lateral sclerosis.

The chief medical advisor for the Muscular Dystrophy Association discussed how his approach to the management and care of patients with neuromuscular disease has been affected by the COVID-19 pandemic.

The phase 3 study will commence following the results of the phase 2b/3 AB10015 study, in which masitinib significantly slowed disease progression in combination with riluzole.

Take 5 minutes to catch up on NeurologyLive's highlights from the week ending April 3, 2020.

Prospective, observational study data suggest that MRS fat fractions and MRI quantitative T2 values can provide disease progression measures in a sensitive and noninvasive manner.

Mayo Clinic is in the forefront of changing practice techniques during the COVID-19 pandemic by using video appointments to reach patients.

NeurologyLive’s parent company, MJH Life Sciences, will be hosting the virtual MJH Life Sciences Sleeves Up Blood Drive from March 31 through April 30.

Neurology News Network for the week ending March 28.

By the end of the open-label extension, 15-17% of patients showed clinically meaningful first response with eculizumab treatment.

Real-world, observational cohort data suggest that Biogen’s nusinersen (Spinraza) is safe and effective in the treatment of spinal muscular atrophy.

Preliminary data from the ASPIRO study of Audentes Therapeutics resamirigene bilparvovec gene therapy suggests it is safe with improvements in neuromuscular and respiratory function.

Patients with spinal muscular atrophy maintained the ability to thrive and achieved motor milestones previously unseen in natural history studies.

Catabasis’s small molecule NF-kB inhibitor showed promise in phase 2, and the phase 3 PolarisDMD study (NCT03703882) of edasalonexant is fully enrolled and expected to read out in late 2020.

In addition to no-charge testing, patients may have access to the Detect Muscular Dystrophy program, a counseling program designed to aid health decisions and follow-up care.

Nearly all patients in this cohort achieved a clinically meaningful >3-point increase during the study period, demonstrating a consistent response to the gene therapy.

Data from the DELOS and SYROS studies of idebenone suggest that investigational DMD treatment has a treatment effect maintained for up to 6 years, which can reduce hospitalizations, bronchopulmonary adverse events, and systemic antibiotic use.

No unexpected safety signals were reported in the post hoc analysis.

Eligible patients with Duchenne muscular dystrophy may receive the drug while it is under priority review by the FDA.

Statistically significant associations between stress-related disorders were identified in those with Alzheimer disease, but not with Parkinson disease or amyotrophic lateral sclerosis.

The chief medical officer of Clene Nanomedicine detailed CNM-Au8, their novel investigational drug currently being assessed in multiple trials for patients with multiple sclerosis and other neurodegenerative diseases.