Risdiplam Gets Prority Review for Presymptomatic Spinal Muscular Atrophy
Risdiplam is a survival motor neuron 2 splicing modifier.
Spinal Muscular Atrophy
Zolgensma Labeling Updated With Additional Safety Information
Zolgensma is an adeno-associated virus vector-based gene therapy.
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.
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.
