Treatment with human fat tissue-derived mesenchymal stem cells (MSCs) may improve motor nerve cell health and survival in infants with spinal muscular atrophy (SMA) type 1, according to data from a small Phase 1 clinical trial in Iran. Notably, the stem cell treatment appeared to have effectively slowed disease progression…
News
The UK SMA Newborn Screening Alliance is calling on people to take action and sign a petition requesting that spinal muscular atrophy (SMA) to be added to the U.K.’s National Health System (NHS) newborn screening program and funded. U.K. citizens and residents can sign the petition, which is collecting…
Many U.S. pediatricians fail to use developmental screening tools and are not familiar with the diagnostic requirements for spinal muscular atrophy (SMA), a study drawing on results of two surveys conducted by Cure SMA reported. These findings, from responses given by hundreds of pediatricians, support a continuing need to…
At its first virtual investor event, biotech company Centogene set a bold mission: to cure 100 rare diseases within the next decade. A leader in the field of genetic diagnostics, Centogene used the June 22 event to present its strategic priorities, outlining its plans to speed the discovery…
Newborn screening for spinal muscular atrophy (SMA) is now offered to 85% of all annual births in the U.S., Cure SMA has announced. The milestone, which means that eight in 10 babies are getting tested at birth, was reached after Texas included the newborn screening on June 1,…
Evrysdi (risdiplam) has been approved in Japan as an oral, at-home treatment of spinal muscular atrophy (SMA). The flavored liquid therapy is also approved in the U.S., Europe, and Canada to treat all types of SMA in patients ages 2 months and older, and its use was favored by regulators…
New data about Zolgensma, the one-time gene therapy for children with spinal muscular atrophy (SMA), demonstrate age-appropriate development when used pre-symptomatically, and rapid, meaningful efficacy in symptomatic children, even those with severe disease before treatment, according to two Phase 3 clinical trials. The therapy, developed by Novartis, uses…
To help patient advocacy leaders and their partners better understand how global health statistics codes — known as ICD codes — are assigned, updated, and revised in the U.S. health information system, the EveryLife Foundation for Rare Diseases is presenting a first-of-its-kind resource guide. The foundation created the…
Newborn screening (NBS) for spinal muscular atrophy (SMA) in Massachusetts allowed for early detection and treatment of affected infants, with most referred to a specialist within the first week of life, three-year data from the state’s SMA screening program show. Notably, the program used a tiered algorithm with screenings that…
SMA News Today is conducting a U.S.-based survey to better understand the characteristics, needs, and treatment experiences of the spinal muscular atrophy (SMA) community. Adults — from patients to clinicians — ages 18 and older are invited to participate. The survey is intended to take no more than 20-25…
