A working group led by Cure SMA, and comprising 15 experts on spinal muscular atrophy (SMA), has developed new guidelines to help clinicians and caregivers decide when to administer therapy to infants with the disease. These come as a response to the recent recommendation by the Advisory Committee on…

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 his thoughts on current SMA therapeutic approaches and care. Darras is a professor of neurology (pediatrics) at Harvard Medical School…

Gyula Acsadi is an expert in three of the world’s most expensive rare illnesses to treat: spinal muscular atrophy (SMA), Duchenne muscular dystrophy (DMD), and Pompe disease. Acsadi, head of neurology and rehabilitation at Connecticut Children’s Medical Center in Hartford, said he’s convinced that new therapies for these…

The Muscular Dystrophy Association (MDA) has asked the medical data services company IQVIA to expand its disease registry into a hub of information on seven neuromuscular conditions, including spinal muscular dystrophy (SMA). The repository will include disease information from care providers, genetics data, and patient-reported information. In addition to SMA, the…

A broad range of clinical studies into AveXis’s gene therapy AVXS-101 is likely to decide whether it is indeed a “transformative” treatment for babies and children with spinal muscular atrophy (SMA) types 1-3. Five trials — ongoing or soon to start worldwide — are planned, including a study in…

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…

Children with spinal muscular atrophy (SMA) type 1, who likely would have died before age 2 only a few years back, were seen  to walk after being treated with Spinraza (nusinersen). This approved therapy, and others with the potential to one day be a treatment option — like a…

Cure SMA recently announced it has committed $5 million in new funding to advance research and care strategies for spinal muscular atrophy (SMA) over the next 12 months. A bit more than half of the total funding will go directly to support local care. The remainder will fund basic…

Cure SMA will host a webinar on spinal muscular atrophy (SMA) today at 1 p.m. EST. The event focuses on access to Spinraza — the only U.S. government-approved therapy to treat the disease, CureSMA said in a press release. Topics to be covered include the current status of dosing in individual…