Oral Myosin Inhibitor Fast Tracked for Becker Muscular Dystrophy
EDG-5506 is a selective, small molecule myosin inhibitor designed to protect injury-susceptible fast skeletal muscle fibers.
Neurologic Disorders
Complaint Prompts FDA Action on Use of Purple Patient Bracelets, Wristbands
The alert comes after a recent complaint about a purple wristband with a different meaning.
Evidence to Support Suicidality Warning for Newer Antiseizure Meds Lacking
The analysis included 5 antiseizure medications (eslicarbazepine, perampanel, brivaracetam, cannabidiol, and cenobamate) evaluated in 17 randomized clinical trials.
Hyperuricemia, Gout Do Not Raise Dementia Risk
Hyperuricemia and gout may be associated with a lower risk for Alzheimer disease.
Botox Approval Expanded to Include 8 New Muscles for Upper Limb Spasticity
The indication for adult upper limb spasticity treatment now includes elbow and forearm muscles, intrinsic hand muscles, and thumb muscles.
Lithium Compared With Newer Therapies as Adjunct to Antidepressants
The likelihood of help or harm, also known as the risk/benefit ratio, was found to be more favorable for lithium.
Stem Cell Therapy Gets Fast Track Status for Parkinson Disease
DA01 is an investigational pluripotent stem cell-derived dopaminergic neuron therapy designed to replace the midbrian dopaminergic neurons lost in Parkinson disease.
Ravulizumab Looks Promising for Generalized Myasthenia Gravis
The randomized, double-blind, placebo-controlled phase 3 study included 175 adults with generalized myasthenia gravis who were not previously treated with a complement inhibitor.
Pridopidine Granted Orphan Drug Status for Amyotrophic Lateral Sclerosis
Pridopidine is an investigational oral drug that binds and activates the sigma-1 receptor, a protein that is highly expressed in the brain.
Janssen COVID-19 Vaccine Fact Sheet Updated With Guillain-Barre Syndrome Warning
The FDA has updated the fact sheet for the Janssen COVID-19 vaccine to include information related to an increased risk of Guillain-Barré syndrome.
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