Mirabegron Granted Priority Review for Neurogenic Detrusor Overactivity
The submissions are supported by data from a phase 3 study that evaluated mirabegron in 91 patients aged 3 to 17 years with NDO on clean intermittent catheterization.
Neuromuscular Disorders
Pregnancy and Delivery Outcomes in Women with Charcot-Marie Tooth Disease
The course of pregnancy and delivery outcomes were not different between women with Charcot-Marie Tooth and the general population, suggesting that the disease may not affect pregnancy.
Safety and Efficacy of Eculizumab in Patients with Refractory Myasthenia Gravis
There was a rapid and sustained clinical response in patients treated with eculizumab for anti-acetylcholine receptor-positive refractory generalized myasthenia gravis.
Trehalose Designated Orphan Drug for ALS
SLS-005 contains the active ingredient trehalose, a low molecular weight disaccharide that crosses the blood brain barrier, stabilizes proteins and activates autophagy.
Investigational Gene Therapy Fast Tracked for Duchenne Muscular Dystrophy
PF-06939926 is a recombinant adeno-associated virus serotype 9 capsid carrying a shortened version of human dystrophin gene under the control of a human muscle-specific promoter.
Casimersen Gets Priority Review for Duchenne Muscular Dystrophy
Casimersen is a phosphorodiamidate morpholino oligomer that works by binding to exon 45 of dystrophin pre-mRNA.
Viltepso Now Available for Duchenne Muscular Dystrophy
Viltepso injection is now available for patients with DMD who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping.
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.
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.
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