Casimersen New Drug Application Submitted for DMD
Sarepta Therapeutics has completed the submission of the casimersen NDA for the treatment of DMD in patients who have genetic mutations that are amenable to skipping exon 45 of the Duchenne gene.
Neuromuscular Disorders
Nusinersen Continues to Improve Presymptomatic Spinal Muscular Atrophy
New data from the phase 2 NURTURE trial evaluating nusinersen demonstrate that presymptomatic patients with spinal muscular atrophy continue to benefit from treatment for up to 4.8 years.
Trial Assessing Efgartigimod for Myasthenia Gravis Meets Primary End Point
Positive topline data was announced from the phase 3 ADAPT trial of efgartigimod (ARG-113; argenx) in adult patients with generalized myasthenia gravis.
Solifenacin Succinate LS Approved for Neurogenic Detrusor Overactivity
The FDA has approved solifenacin succinate LS oral suspension for the treatment of neurogenic detrusor overactivity in pediatric patients 2 years of age and older.
FDA Grants Fast Track Status to Arimoclomol for ALS
The FDA has granted Fast Track designation to arimoclomol (Orphazyme A/S) for the treatment of amyotrophic lateral sclerosis.
Insights Into the Diagnosis and Management of Amyotrophic Lateral Sclerosis
A diagnosis of amyotrophic lateral sclerosis can be overlooked by clinicians unfamiliar with the various signs of the disease.
Guidance for Management of Myasthenia Gravis and Lambert-Eaton Myasthenic Syndrome During COVID-19
The International Myasthenia Gravis/COVID-19 Working Group has published a short guidance statement on the management of MG and Lambert-Eaton myasthenic syndrome during this novel pandemic.
Benefits on Quality of Life With Nusinersen for Spinal Muscular Atrophy
In patients with SMA, treatment with nusinersen was associated with improvement in health-related quality of life and reduced impact on caregivers.
Synthetic Flavanol Gets Orphan Drug Status for Duchenne and Becker Muscular Dystrophy
The FDA has granted Orphan Drug designation to EB 002 ((+)-epicatechin; Epirium Bio) for the treatment of Duchenne and Becker muscular dystrophy.
New Data Show Continued Benefit With Onasemnogene Abeparvovec-xioi in Spinal Muscular Atrophy
Patients with spinal muscular atrophy treated with a one-time infusion of onasemnogene abeparvovec-xioi continue to show clinically meaningful benefits even 5 years after dosing.
-
Loading...
-
Loading...
Next post in Neuromuscular Disorders
