Apitegromab Fast Tracked for Spinal Muscular Atrophy
Apitegromab is a selective inhibitor of the inactive form of myostatin to block its activation in the muscle.
Spinal Muscular Atrophy
Long-Term Data Demonstrate Sustained Efficacy of Risdiplam in Spinal Muscular Atrophy
In Part 2 of the trial, patients 2-25 years of age received either risdiplam for 24 months or placebo for 12 months followed by risdiplam for 12 months.
Zolgensma Benefits Observed in Presymptomatic SMA Patients
The phase 3 SPR1NT trial is an ongoing open-label, single-arm study evaluating onasemnogene in presymptomatic patients (≤6 weeks of age) with a genetic diagnosis of SMA and 2 or 3 copies of survival motor neuron 2 gene.
MPR Weekly Dose Podcast Episode 47
This week we have remdesivir news; A new opioid and spinal muscular atrophy treatments are approved; A sublingual epinephrine treatment; And the latest on Biogen’s investigational Alzheimer disease treatment. Also, we have a brief summary of the other drugs in the pipeline from the past week.
Oral Therapy Evrysdi Approved for Spinal Muscular Atrophy
Evrysdi is a survival motor neuron-2 (SMN2) splicing modifier.
Nusinersen Continues to Benefit SMA Patients Treated Presymptomatically
New data from the phase 2 NURTURE trial evaluating nusinersen (Spinraza®; Biogen) demonstrate that presymptomatic patients with spinal muscular atrophy (SMA) continue to benefit from treatment for up to 4.8 years.
New Data Show Continued Benefit With Onasemnogene Abeparvovec-xioi in Spinal Muscular Atrophy
Patients with spinal muscular atrophy (SMA) treated with a one-time infusion of onasemnogene abeparvovec-xioi (Zolgensma®;AveXis) continue to show clinically meaningful benefits even 5 years after dosing.
SUNFISH: Motor Function Improvement With Risdiplam in Type 2 or 3 Spinal Muscular Atrophy
Risdiplam is an investigational, orally administered liquid survival motor neuron-2 (SMN2) splicing modifier designed to increase and sustain SMN protein levels throughout the central nervous system and peripheral tissues of the body.
Risdiplam Gets Priority Review for Spinal Muscular Atrophy
Recently announced results from part 2 of the SUNFISH trial, which evaluated the safety and efficacy of risdiplam in patients with type 2 or 3 SMA aged 2 to 25 years, showed that the treatment met its primary end point.
Risdiplam Meets Primary End Point in Motor Function for Spinal Muscular Atrophy
Genentech announced positive data from the pivotal part 2 of the SUNFISH trial that evaluated the safety and efficacy of risdiplam in adult and pediatric patients with Type 2 or Type 3 spinal muscular atrophy (SMA).
