Neuromuscular

The effect observed in on ALSFRS-R at week 48 and progression-free survival further support the premise of greater treatment effect when masitinib is initiated earlier in the disease.

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John Brandsema, MD, comments on the importance of patient compliance to therapy to manage spinal muscular atrophy.

Experts in pediatric neurology share insights on the role of small molecule treatment for SMA through the recent FDA approval of risdiplam and discuss factors to consider when tailoring therapy for individual patients.

Take 5 minutes to catch up on NeurologyLive's highlights from the week ending July 15, 2021.

Since vaccine administration, the CDC has announced 100 preliminary cases of Guillain-Barré syndrome.

The chief medical officer of Scholar Rock described how the investigational apitegromab aims to fill some of the current unmet needs for patients with spinal muscular atrophy.

Efficacy results from a 109-week interim analysis showed improvement for patients treated with viltolarsen when compared to the matched DMD historical control group.

The chief medical officer of Scholar Rock detailed the additive benefits apitegromab has in treating SMA when used with previously approved nusinersen.

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John Brandsema, MD, leads the discussion on the concept of gene transfer for the management of SMA and the recent approval of onasemnogene abeparvovec-xioi.

Experts in pediatric neurology provide insight on the efficacy of nusinersen and treatment response in patients with SMA.

Neurology News Network for the week ending July 10, 2021.

Take 5 minutes to catch up on NeurologyLive's highlights from the week ending July 9, 2021.

Suggestions from Mary Anne Ehlert, founder and president, Protected Tomorrows, include getting involved with the community and making the most of benefits and programs.

Advocacy organization Cure SMA recap their recently concluded meeting, with notable presentations and medical education sessions led by world leaders.

The chief medical officer of Scholar Rock provided background on the company’s investigational SMA treatment and its clinical benefit when applied with nusinersen (Spinraza; Biogen).

The chief medical officer of Scholar Rock discussed topline results from the phase 2 TOPAZ study of apitegromab in patients with spinal muscular atrophy.

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Take 5 minutes to catch up on NeurologyLive's highlights from the week ending July 2, 2021.

The manuscript was published in the May 2021 issues of BMC Pediatrics.

John Brandsema, MD, and Julie Parsons, MD, discuss the approval of nusinersen for the treatment of spinal muscular atrophy and their personal experiences regarding drug safety and administration in patients.

Expert pediatric neurologists review the importance of early recognition and referral to neurologists for the diagnosis of spinal muscular atrophy and when it’s appropriate to approach treatment.

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