Noninvasive Interventions Can Reduce Spine Pain Versus Usual Care
Small reductions in pain-related disability at three months for multidisciplinary biopsychosocial intervention, individualized postural therapy
Diagnosis & Disease Information
Opioid Rx Dosage Higher for Adults With Cerebral Palsy, Spina Bifida
Higher prescription of oral morphine equivalents seen for adults with cerebral palsy, spina bifida across pain categories
Risdiplam Gets Prority Review for Presymptomatic Spinal Muscular Atrophy
Risdiplam is a survival motor neuron 2 splicing modifier.
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.
