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.
The entire 25-patient cohort from the NURTURE study was able to sit without support, while 88% were able to walk independently, according to new 45-month data in presymptomatic patients with spinal muscular atrophy.
PF-06939926 is a recombinant adenoassociated virus serotype 9 (AAV9) capsid carrying mini-dystrophin, a shortened version of the human dystrophin gene, under the control of a muscle-specific promoter.
The clinical assistant professor of neurology and neurosurgery at the University of Texas spoke about the staggering number of men who forgo seeing physicians for serious conditions and how the clinical community can help address the challenge.
Neurology News Network for the week ending June 22, 2019.
The FDA has approved an sBLA for deflazacort that expands the label to patients aged 2 to 5 with Duchenne muscular dystrophy.
Zilucoplan, also being developed for generalized myasthenia gravis, is expected to enter into a phase 2 clinical trial in IMNM in the second half of 2019, according to Ra Pharma.
The FDA designation was granted to the SK channel modulator after it showed promise in preclinical and phase 1 studies. Cadent Therapeutics is planning a phase 2a trial for the latter half of 2019.
Neurology News Network for the week ending June 1, 2019.
