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April 20, 2018April 20, 2018

#AAN2018 — Spinraza Improves Motor and Respiratory Function in SMA Children, Trials Show

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Spinraza (nusinersen) improves motor and respiratory function in children with spinal muscular atrophy (SMA), according to results from four trials. Biogen will present the data at the American Academy of Neurology annual ... Read more

April 18, 2018

#AAN2018: SMA Treatment Candidate RG7916 Increases Amount of Key SMN Protein, Clinical Trials Show

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Roche’s treatment candidate RG7916 increased the amount of full-length, functional SMN protein in spinal muscular atrophy (SMA) patients, according to soon-to-be-presented data from a Phase 2/3 clinical trial. The company will ... Read more

April 16, 2018April 17, 2018

#AAN2018 – Olesoxime Stabilizes SMA Patients’ Long-term Movement Function, Extension Study Shows

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Olesoxime (TRO19622) stabilizes the movement function of patients with Type 2 or Type 3 spinal muscular atrophy over the long term, an extension of a Phase 2 clinical trial ... Read more

April 13, 2018April 13, 2018

Researchers Discover Promising New Gene Therapy Approach for Spinal Muscular Atrophy

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A new genetic approach to restore the expression of the gene that causes spinal muscular atrophy (SMA) in patients’ cells holds promise to effectively and permanently treat the disease, ... Read more

April 12, 2018April 12, 2018

NIH Agency Pioneers Collaborative Research into Rare Diseases

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A little-known government entity within the National Institutes of Health (NIH) is helping to lead U.S. efforts to speed up the development of therapies for some 7,000 rare diseases. ... Read more

April 11, 2018April 11, 2018

Muscle Waste Product Creatinine Might Be Used as SBMA Biomarker, Study Reports

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Blood levels of a waste product from muscle metabolism could be used to see how spinal and bulbar muscular atrophy (SBMA) develops before symptoms appear, a Japanese study reports. The ... Read more

April 9, 2018April 9, 2018

Novartis to Acquire AveXis and Its SMA Gene Therapy for $8.7B in Cash

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AveXis, which is running a series of pivotal clinical trials testing a gene therapy in babies and children with spinal muscular atrophy (SMA), announced plans to be acquired by ... Read more

April 8, 2018April 9, 2018

SMA Expert Reveals Insights into Patient Care, Hope for Future Cure in Interview

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Spinal muscular atrophy (SMA) expert Basil T. Darras, MD, is optimistic about treatment advances and the possibility of a future cure, according to a recent interview in which he shared ... Read more

April 6, 2018April 6, 2018

FDA Grants Orphan Drug Status to SRK-015 for SMA

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SRK-015 has received orphan drug status designation by the U.S. Food and Drug Administration (FDA) to treat muscle atrophy in patients with spinal muscular atrophy (SMA). SRK-105 is Scholar Rock’s lead product ... Read more

April 4, 2018April 4, 2018

AveXis’ SMA Type 1 Gene Therapy Granted Speedier Review in Japan

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AVXS-101 has been awarded the SAKIGAKE designation in Japan for the treatment of spinal muscular atrophy (SMA) type 1. This designation will speed the therapy’s development and review process and allow ... Read more

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