Neuromuscular

Neurology News Network for the week ending October 12, 2019.

The impact MT1621 had on survival probably for those treated in comparison to the natural history of the often-fatal disorder was highly significant, with all patients remaining alive as of October.

Treatment with the Catabasis Pharmaceuticals small molecule oral NF-kB inhibitor resulted in improvements in the 10-meter walk/run scores, time-to-stand scores, and 4-stair climb scores in 31 steroid-naïve boys with DMD aged 4 to 7 years.

Neurology News Network for the week ending October 5, 2019.

New 9-month data suggest that Sarepta Therapeutics’ investigational limb-girdle muscular dystrophy type 2E treatment, SRP-9003, is linked to improved functional measures and reduction in creatine kinase.

The senior research scientist in the Center for Mobility and Rehabilitation Engineering Research at Kessler Foundation spoke to the research being done into the functional implementation of exoskeletons in neurorehabilitation.

The drug, the only approved botulinum toxin type B on the market, was recently approved for the treatment of chronic sialorrhea in adults.

Neurology News Network for the week ending September 21, 2019.

A prospective, cross-sectional assessment suggests that patients with DMD can be adequately assessed for cognition in a brief period of time using the NIHTB‐CB, and additionally suggested a cognitive vulnerability in mothers who were carriers of DMD mutation.

Further analysis of the SHINE trial suggest nusinersen is linked to 6-year maintained improvement in patients with SMA. Biogen has announced a new global trial of a 50 mg loading dose in a broader cohort.

The 1st Annual Congress on the Future of Neurology will take place September 27-28, 2019 at the InterContinental New York Times Square in New York City.

Wave Life Sciences intends to file for accelerated approval in the second half of 2020 pending muscle biopsy results from an open-label extension study.

Ra Pharmaceuticals’ macrocyclic peptide inhibitor of complement component 5 is expected to enter phase 3 of its development in the latter half of 2019 after success in a phase 2 trial of its potential in myasthenia gravis.

Neurology News Network for the week ending September 7, 2019.

Serum NfL levels, at as early as the time of diagnosis, were a strong and independent predictor of death in patients with amyotrophic lateral sclerosis, with the study results expanding on the findings of prior literature.

US WorldMeds rimabotulinumtoxinB injection showed significant decreases in sialorrhea symptoms with a single treatment as soon as the first week, lasting up to 3 months in clinical studies.

Neurology News Network for the week ending August 24, 2019.

The child neurology resident at NYU Langone spoke about ways for residents to identify and address impaired colleagues that suffer from depression and burnout.

The regulatory agency cited 2 concerns in the complete response letter: the risk of infections related to intravenous infusion ports and renal toxicity.

The investigational candidate is the subject of a phase 3 study assessing efficacy and safety in adults with hATTR amyloidosis with transthyretin mutation.

The associate chief of the MS division and professor of neurology at the University of Pennsylvania described the relationship between artificial intelligence and medicine, and how he sees it evolving in the future.

The postdoctoral scholar at the University of California San Francisco discussed the findings from her study that gives comprehensive insight into prevalence and organ associations of vasculitic neuropathy.

Neurology News Network for the week ending August 10, 2019.

The Rare Disease Cures Accelerator-Data and Analytics Platform, aimed at accelerating the development of new therapies, will launch at a joint meeting between the Critical Path Institute and the National Organization for Rare Disorders on Tuesday, September 17 in Bethesda, Maryland.

The agency said the gene therapy should remain on the market while it assesses the situation and does not impact their evaluation of data from the human clinical trials.