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FDA Action Update, April 2024: Approvals, Clearance, and Complete Response Letter
The FDA was busy in April 2024, making a number of decisions on potential new therapeutic agents including granting approvals, a clearance, and a fast track designation, issuing a...
Cell Therapy Reveals Early Promise in Addressing Motor and Non-Motor Symptoms for Parkinson Disease
Claire Henchcliffe, MD(Credit: Weill Cornell Medicine)According to a recent report from GlobalData, using data from BlueRock Therapeutics’ phase 1 study (NTC04802733) assessing be...
NeuroVoices: Kayla Scippa, on Recognizing Unmet Need in Chronic Demyelinating Polyneuropathy
Chronic demyelinating polyneuropathy (CIDP) is a neuromuscular disorder characterized by progressive weakness and impaired sensory function in the arms and legs. It is an autoimmun...
SAGE-324/BIIB124 Significantly Reduces Upper Limb Tremor in Phase 2 KINETIC Trial
Helen Colquhoun, MD(Credit: LinkedIn)Recently published in Movement Disorders, results from the double-blind, randomized, placebo-controlled, phase 2 KINETIC trial (NCT04305275) as...
AEON Biopharma Reports Disappointing Interim Phase 2 Results of Migraine Preventive ABP-450
Newly announced preliminary top-line results from a planned interim analysis of a phase 2 study (NCT04845178) showed that treatment with ABP-450 (AEON Biopharma) did not meet its p...
NeurologyLive® Friday 5 — May 10, 2024
Welcome to NeurologyLive®'s Friday 5! Every week, the staff compiles 5 highlights of NeurologyLive®'s widespread coverage in neurology, ranging from newsworthy study findings and...
ALS Candidate PrimeC Shows Greater Treatment Effect in High-Risk ALS
Jeremy M. Shefner, MD, PhDNewly announced analyses from the phase 2b PARADIGM trial (NCT05357950) showed that treatment with PrimeC (NeuroSense), an investigational agent made up o...
Negative Myoclonus Associated With Severe Motor Implications in Progressive Myoclonus Ataxia
Marina A.J. Tijssen, MD, PhD(Credit: University of Groningen)In a newly published study in Movement Disorders, patients with progressive myoclonus ataxia (PMA) affected by negative...
Crossover Study Highlights Pharmacokinetic Effects of DHE Powder STS101
A recently published, randomized, open-label, 5-period crossover study showed that treatment with STS101, an intranasal dihydroergotamine (DHE) powder, was safe in healthy particip...
Patient Death Reported in Phase 2 DAYLIGHT Study of Pfizer’s Gene Therapy for Duchenne
A patient in the phase 2 DALIGHT trial (NCT05429372) assessing Pfizer’s investigational gene therapy fordadistrogene movaparvovec (PF-06939926) in boys with Duchenne muscular dyst...