Investigational Limb-Girdle Muscular Dystrophy Treatment Shows Promise at 9 Months
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.
Dysphagia is a common symptom and complication of neurologic disorders, yet no clear treatment protocol has been identified.
The staff neurologist at Cleveland Clinic’s Mellen Center for MS shared her insight into the use of telemedicine in an outpatient setting across a number of subspecialties in neurology and how it can supplement care going forward.
The phase 2b trial of the repository corticotropin injection (Acthar Gel) in amyotrophic lateral sclerosis was halted due to data and safety board concerns of the potential risk of pneumonia.
The complete response letter cited issues with an active pharmaceutical ingredient used in the Biohaven 2017 bioequivalence study that was manufactured between 2014 and 2016 in an Apotex facility.
The International Congress on the Future of Neurology will take place this Fall in New York City, converging experts in neurology to discuss the latest data and best practices to better inform clinical decision-making.
Neurology News Network for the week ending July 6, 2019.
Preclinical trials and success stories suggest that much is riding on vector-based therapies for the treatment of rare neurological conditions.
The clinical assistant professor of neurology and neurosurgery at the University of Texas discussed the need to identify men who may be at risk for myasthenia gravis despite a lack of willingness to see their physician.
