Alex Coletta,  —

Cure SMA is granting $450,000 to help increase capacity at spinal muscular atrophy (SMA) sites across the United States. The money — with $50,000 to be given to nine sites — aim to boost the number of patients that sites can follow, treat and monitor. Five of the…

Throughout 2017, SMA News Today has brought you daily coverage of spinal muscular atrophy (SMA)-related advocacy events, clinical studies and research updates. These were the top 10 SMA news stories of 2017, ranked according to the number of views each story received. No. 10 – AveXis’ Gene Therapy…

Biogen and Ionis Pharmaceuticals, the companies that developed the new spinal muscular atrophy therapy Spinraza (nusinersen), will continue their partnership under a new agreement. “We are pleased to extend our collaboration with our valued colleagues at Ionis, which we believe complements our ongoing efforts to enhance and build…

Spinal muscular atrophy advocates have asked American regulatory officials to add SMA to the list of genetic conditions the federal government thinks newborns should be screened for. The request came in a meeting between the advocates and the government’s Advisory Committee on Heritable Disorders in Newborns and Children on Nov. 8.

A Phase 3 clinical trial to evaluating the safety and efficacy of an investigational gene replacement therapy by AveXis is now recruiting infants with spinal muscular atrophy type 1. The trial of AVXS-101 is seeking patients younger than six months of age. Participants must have a genetic mutation analysis confirming SMA type 1 diagnosis, according to a news release from the Muscular Dystrophy Association. AVXS-101 is designed to specifically deliver the fully functional human SMN gene to motor neurons, which SMA patients lack. This will restore normal levels of survival motor neuron protein in these nerve cells, allowing them to properly control muscle activity and improve strength and function. Results of a Phase 1 study showed that the motor functions of babies with SMA type 1 show clinically meaningful improvements after one single intravenous infusion. Eight of the 15 infants treated with AVXS-101 were able to sit without assistance and two could crawl, stand or walk independently — all abilities never seen in untreated SMA infants. The U.S. Food and Drug Administration has granted AVXS-101 Orphan Drug Designation to treat all types of SMA. AVXS-101 has also received Breakthrough Therapy Designation and Fast Track Designation to treat SMA Type 1. Both will speed up the drug's clinical development and potential approval. The STR1VE study is an open-label Phase 3 trial to evaluate the impact of AVXS-101 on children’s development and overall survival. It will likely include 15 infants with genetically confirmed non-functional SMN1 gene, but with one or two copies of the SMN2 gene. The study — to be conducted at clinics in California, Colorado, Illinois, Maryland, New York, Ohio and Oregon — will evaluate patients' capacity to sit by themselves at 18 months of age, as well as their ability to breathe without additional support upon receiving one injection of AVXS-101. All required clinical visits, tests and additional treatments will be provided to participants at no cost, as well as travel assistance for families who don't live near any of the study sites. For additional information on the STR1VE trial, please visit the study website or the study registry page. To participate, contact the trial coordinator at the nearest site.

Spinraza (nusinersen) was granted the 2017 Prix Galien USA Award for Best Biotechnology Product in recognition of its achievement in scientific innovation for the treatment of spinal muscular atrophy (SMA), Biogen and Ionis announced. The award was presented at a ceremony in New York City,…

Texas is considering adding spinal muscular atrophy (SMA) to its list for newborn screening in the wake of the U.S. Food and Drug Administration’s approval in December of Spinraza (nusinersen), the first treatment approved for a broad range of SMA patients. The addition of SMA to Texas’ newborn screening list…

With August recognized as SMA Awareness Month, this year the spinal muscular atrophy (SMA) community is focused on promoting nationwide newborn screening. Since 1996, Cure SMA has coordinated efforts to give the SMA community a specific time of year to think about the latest advancements and to balance what still…

Spinraza (nusinersen) was just approved by Health Canada for the treatment of 5q spinal muscular atrophy (SMA) patients, Biogen Canada announced. This is the first therapy approved by Health Canada for the treatment of SMA. The drug is specifically indicated to treat 5q SMA, which represents about…

Cytokinetics will present the latest developments on its drug candidate CK-2127107, also known as CK-107, for spinal muscular atrophy (SMA) during the Cure SMA 2017 Annual SMA Conference June 29 to July 2 in Orlando, Florida. Developed in collaboration with Astellas, CK-107 was designed to increase muscle function and…