Matt Hoffman, Associate Editorial Director for NeurologyLive®, has covered medical news for MJH Life Sciences, NeurologyLive®’s parent company, since 2017. He hosts the NeurologyLive® Mind Moments® podcast, as well as the Medical World News show, Deep Dive. Follow him on Twitter @byMattHoffman or email him at mhoffman@neurologylive.com
AT-GAA’s Conceivable Benefits for Pompe Disease and the Need for More Options
September 9th 2022Barry J. Byrne, MD, PhD, the associate chair of pediatrics and director of the Powell Gene Therapy Center at the University of Florida; and the chief medical advisor to the Muscular Dystrophy Association, offered his perspective on the upcoming FDA decision on AT-GAA (Amicus Pharma).
Insomnia Alone Linked to Worsened Symptoms and Impairments Than OSA Alone in Military Personnel
September 8th 2022Similarly, the prospective observational data of active duty military individuals showed that comorbid obstructive sleep apnea and insomnia resulted in worsened symptoms and sleep-related impairments.
FDA Lifts Hold on Phase 2 Trial of SRP-5051 in DMD Amenable to Exon 51 Skipping
September 6th 2022The hold was originally placed on part B of the phase 2 MOMENTUM trial in Duchenne muscular dystrophy following a June 2022 report of hypomagnesemia after treatment with high-dose SRP-5051. Going forward, Sarepta will adjust its global trial protocol.
Sirolimus Topical Gel Available for Facial Angiofibroma Associated With Tuberous Sclerosis Complex
August 30th 2022The 0.2% topical gel, branded as Hyftor by Nobelpharma America, was approved in March 2022, and is indicated for individuals aged 6 years and older with TSC. It is the first topical therapy approved for this population.
FDA Approves Midazolam Autoinjector for Status Epilepticus
August 30th 2022The Rafa Laboratories 10-mg midazolam autoinjector is the first in market product of its kind, adding another option for the rescue of individuals with status epilepticus, a condition with crucial time implications in treatment.
Phase 2 Trial of OXE103 in Concussion Reports Positive Interim Progress
August 18th 2022The investigational therapy is a twice-daily injectable identical formulation of the protein ghrelin, being assessed in a 2-part trial including individuals who are highly symptomatic within 28 days of brain injury.
Long-term Immunosuppression Treatment Is Suitable for Patients With NMOSD
August 16th 2022Data suggest that long-term treatment with immunosuppression therapies, such as rituximab, is generally appropriate for patients with neuromyelitis optica, though transverse myelitis might indicate a higher risk of relapse post discontinuation.
Passage Bio Doses First Patient in Frontotemporal Dementia Gene Therapy Trial
August 16th 2022The AAV1 gene therapy, PBFT02, is being assessed in a phase 1/2 trial called upliFT-D (NCT04747431), which is planned to take place over the course of 24 months in patients with frontotemporal dementia with granulin mutations.
CNM-Au8 Displays Positive Topline Results in Multiple Sclerosis, Supportive of Phase 3 Trial
August 16th 2022Clene Nanomedicine’s gold nanocrystal suspension treatment showed significant improvements in Low Contrast Letter Acuity and Multiple Sclerosis Functional Composite scores among individuals with relapsing multiple sclerosis.
Alnylam Reports Positive Data on Patisiran in APOLLO-B Trial of ATTR Amyloidosis With Cardiomyopathy
August 4th 2022The Alnylam Pharmaceuticals agent, already approved for the treatment of polyneuropathy in those with hATTR amyloidosis, has now shown promise as a therapeutic option for patients with ATTR amyloidosis with cardiomyopathy.