SMA Type I

May 21, 2018June 7, 2018

#CureSMA 2018 – Results of Phase 2 Test of Reldesemtiv in 70 SMA Patients Set for Release at Meeting

News

Results of an exploratory Phase 2 study evaluating the effectiveness of reldesemtiv (CK-2127107) for a first time in patients with spinal muscular atrophy (SMA) will be presented at the Cure SMA annual conference set ... Read more

April 4, 2018April 4, 2018

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

News

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

July 7, 2017image

How Is SMA Diagnosed?

Spinal muscular atrophy (SMA) is a genetic neuromuscular disease. SMA patients have either a missing or altered gene, (the survival motor neuron-1 or SMN1 gene), which causes large motor nerves ... Read more

June 22, 2017image

SMA Explained: 15 Keywords You Should Know

Becoming a parent is always an overwhelming and sometimes daunting experience. This is amplified when you discover that your child has a health condition like spinal muscular atrophy (SMA). Cure SMA ... Read more

September 29, 2016

Children with SMA May Also Show Symptoms of Farber Disease, Case Study Reports

News

Children with spinal muscular atrophy (SMA) may present symptoms indicative of other genetic disorders, such as Farber disease, according to a case study. Researchers also highlighted the importance of analyzing ... Read more

September 27, 2016September 27, 2016

Biogen Completes Submission of New Drug Application for SMA Therapy Nusinersen

News

Biogen has completed its rolling submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for the approval of nusinersen for the treatment for spinal muscular ... Read more

September 8, 2016September 27, 2016image

The 4 Types of Spinal Muscular Atrophy

Spinal muscular atrophy (SMA) is a debilitating, severe disease that affects mainly children, shutting down their body, their muscles and organs but in varying ways according to type. There ... Read more

March 29, 2016March 29, 2016

AveXis Releases Favorable Data in Ongoing Phase 1 Test of SMA Gene Therapy, AVXS-101

News

AveXis recently reported favorable results from an ongoing Phase 1 clinical trial examining its candidate, AVXS-101, for the systematic delivery of gene therapy to young patients with spinal muscular atrophy (SMA). These preliminary ... Read more

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