Five states — Missouri, Minnesota, New York, Pennsylvania and Utah — now routinely screen newborn babies for spinal muscular atrophy (SMA), while another 13 have passed laws either requiring such screening or are in the process of doing so. Pennyslvania began testing infants for the neuromuscular disease on March 1,…
Scotland has joined the growing roster of countries making the spinal muscular atrophy (SMA) therapy Spinraza available to patients at low or no cost through that country’s public health system. Beginning in April, all SMA patients using NHS Scotland may start to receive Spinraza injections. Clearing the path for…
Researchers have developed a platform called SMArtCARE that allows prospective monitoring of all spinal muscular atrophy patients regardless of their current treatment. The technology has the potential to shed new light on the disease’s natural history and medication’s influence. A description of the project was published in…
Visitors to the Clinic for Special Children (CSC) just outside Strasburg, Pennsylvania, might be forgiven for thinking they’ve made a wrong turn. Hidden at the end of a long, winding driveway off Bunker Hill Road, the clinic, which serves the Amish and Mennonite communities of Lancaster County and beyond, looks…
The American Society of Gene and Cell Therapy (ASGCT) has unveiled a new online resource designed to keep patients, caregivers, and the public apprised of the latest in gene and cell therapies, including those intended for spinal muscular dystrophy (SMA). Gene Therapy 101 is the organization’s debut…
SMA News Today brought you daily coverage of important findings, key treatment developments and clinical trials related to spinal muscular atrophy (SMA) throughout 2018. We look forward to reporting more news that is important for the SMA community during 2019. Here are the top 10 most-read SMA articles of 2018, with…
A few days ago, I met with an occupational therapist to evaluate the effects of my Spinraza (nusinersen) treatments. So far, I have had six injections of this therapy, and I have noticed things like increased energy and flexibility in my muscles. Still, I don’t…
LMI070 (branaplam), a candidate oral therapy under development by Novartis, is being tested in a Phase 1/2 clinical trial for infants with spinal muscular atrophy (SMA) type 1. A recent study describes how the compound was discovered from a large screen for SMN2…
Novartis may put a $4 million to $5 million price tag on its “one-time, potentially curative” gene therapy AVXS-101 for spinal muscular atrophy (SMA) and believes this could be cost-effective. “Four million dollars is a significant amount of money, but we believe that this is a cost-effective point,”…
Preliminary data from two ongoing clinical trials into risdiplam (RG7916) — a potential oral therapy for all types of spinal muscular atrophy — support its considerable promise, with treated babies now able to “roll, sit, kick … things that they [otherwise] never do,” the CEO of PTC Therapeutics said in…