Edasalonexent Slows DMD Progression in Phase 2 Trial
October 7th 2019Treatment 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.
Investigational Limb-Girdle Muscular Dystrophy Treatment Shows Promise at 9 Months
October 5th 2019New 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.
Bernard Baumel, MD: Evaluating Stem Cells in Alzheimer Disease
October 4th 2019Bernard Baumel, MD, and colleagues at the University of Miami have recently launched a clinical trial to examine the safety and efficacy of multiple doses of human mesenchymal stem cells in patients with Alzheimer disease.
Marie D'hooghe, MD, PhD: Pregnancy Plans for Patients With MS
October 4th 2019The neurologist at the National MS Center and University Hospital, in Brussels, discussed the clinician mindset when treating women with MS who plan to get pregnant, and how patients using assistive reproductive treatments should be approached.
Acthar Gel Shows Top-Line Success in MS Relapse Registry
October 3rd 2019Mallinckrodt’s repository corticotropin injection (Acthar Gel) showed statistically significant results for every efficacy end point assessed, including the primary end point. Data will be shared at an upcoming medical meeting.
Upsher-Smith Launches Sumatriptan Nasal Spray for Acute Migraine Treatment
October 2nd 2019Originally approved by the FDA in January, sumatriptan nasal spray was acquired by Upsher-Smith in a territory rights agreement with Dr. Reddy’s Laboratories in June, as well as its 3-mg injection formulation (Zembrace SymTouch).
Karen J. Nolan, PhD: Functional Implementation of Robotics in Neurorehabilitation
October 1st 2019The 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.
Gut Microbiota: The Key to Parkinson Disease?
October 1st 2019The professor of chemistry and chemical biology at Harvard University and Blavatnik National Laureate discussed the work she and colleagues have done on microbiome metabolism and what upcoming plans to pursue this further could achieve.
Lemborexant Proves Safe, Effective for Insomnia in Long-Term Phase 3 Study
September 30th 2019Year-long data presented at World Sleep 2019 suggest that Eisai’s lemborexant is safe and effective in treating adults with insomnia, supporting its recently submitted NDA. It is set to be reviewed by the FDA by late December.
CGRP: Paving the Path to Migraine and Its Treatment
September 30th 2019After a long period of time with minimally effective treatments for migraine, the growing understanding of the CGRP pathway has opened the door to a variety of new and highly efficacious therapies for patients with chronic and episodic forms of disease.
JZP-258 Shows Success In Stabilizing Cataplexy for Patients With Narcolepsy
September 28th 2019Data presented at World Sleep 2019 has suggested that the investigation Jazz Pharmaceuticals sodium oxybate agent, JZP-258, is efficacious and safe in treating patients with narcolepsy who have cataplexy.
AbobotulinumtoxinA Adds Upper Limb Spasticity to Pediatric Indication, Excluding Cerebral Palsy
September 27th 2019This regulatory approval in the treatment of pediatric patients aged 2 years and older with upper limb spasticity, excluding those with cerebral palsy, marks abobotulinumtoxinA (Dysport, Ipsen) as the first botulinum toxin approved for both upper and lower pediatric spasticity.
Karen J. Nolan, PhD: Translational Research Into Neurorehabilitation Exoskeletons
September 27th 2019The senior research scientist in the Center for Mobility and Rehabilitation Engineering Research at Kessler Foundation shared insight into the work she and her colleagues are doing to try to revolutionize the way exoskeletons can improve neurorehabilitation and gait challenges.
Zogenix Resubmits Fenfluramine NDA in Dravet Syndrome
September 26th 2019Following the receipt of a refusal to file letter from the FDA, Zogenix has resubmitted its application for fenfluramine hydrochloride (Fintepla), seeking approval for the treatment of seizures associated with Dravet syndrome.