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See below for a daily digest of the latest SMA news!

Patients, Supporters Worldwide Recognizing Rare Disease Day 2020

Feb 21, 2020 01:53 pm | Mary Chapman

 

RareAn abundance of events are afoot around the world to mark Rare Disease Day 2020 on Feb. 29. The activities are focused on heightening awareness about rare diseases and the hundreds of millions of individuals they are thought to affect. Patients, caregivers, and advocates worldwide will sport denim ribbons and zebra stripes, paint their faces, […]

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SRK-015 Data Continue to Support Potential for SMA, Scholar Rock Says

Feb 21, 2020 09:00 am | Patricia Inacio, PhD

 

SRK-015Preclinical and clinical data on SRK-015 continue to support the therapy’s potential to treat spinal muscular atrophy (SMA), developer Scholar Rock said in a press release. Recruitment for a Phase 2 clinical trial evaluating the efficacy and safety of SRK-015 in children and adults with SMA type 2 and type 3 is complete, the company […]

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Risdiplam Data to New Way of Classifying ‘Types’ Among SMA Europe 2020 Highlights, Advocate Says

Feb 19, 2020 08:00 am | Marisa Wexler, MS

 

SMA Europe conferenceResults from a clinical trial of risdiplam in a broad range of people with spinal muscular atrophy were a highlight of SMA Europe’s 2020 conference, a board member of that SMA umbrella patient advocacy group said in an interview. Also of interest were discussions about a possible paradigm shift in how SMA is classified, Kacper […]

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Risdiplam Use ‘Meaningful’ to Broadest SMA Group Ever in Clinical Trial, Genentech Exec Says

Feb 18, 2020 10:00 am | Marta Figueiredo

 

risdiplamRisdiplam treatment has led to “clinically meaningful” improvements — whether gains or disease stabilization — in the broadest range of spinal muscular atrophy (SMA) patients, by age and disability level, yet enrolled in a clinical trial. More than one-third of those in the second part of the SUNFISH trial (NCT02908685) were ages 12 or older — […]

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