Jerry R. Mendell, MD, has been named to the inaugural TIME100 Health list for his decades-long contributions to the treatment of neuromuscular diseases — such as spinal muscular atrophy (SMA) and Duchenne muscular dystrophy (DMD) — and the advancement of gene therapy. The global media brand’s list…
The Muscular Dystrophy Association (MDA) has chosen Ira Walker, a Florida resident living with spinal muscular atrophy (SMA) type 2, as this year’s national ambassador. In this new role, the 39-year-old Walker will share his story nationwide, with an aim of raising awareness of the MDA’s mission…
A new children’s book — one whose story is inspired by the author’s own family — seeks to heighten awareness of spinal muscular atrophy (SMA) while helping young readers appreciate and understand differences in others. Written by Alvarys Santana, the 34-page book, titled “My Sister is Special,”…
The Muscular Dystrophy Association (MDA) has opened the MDA Gene Therapy Support Network — a new program it’s dubbed GTx — to aid and educate families living with spinal muscular atrophy (SMA) or other neuromuscular diseases (NMDs) who may be considering a gene therapy as part of…
The Tufts Medical Center, a teaching hospital of the Tufts University School of Medicine, has been awarded a Muscular Dystrophy Association (MDA) grant to investigate patient access to treatments for spinal muscular atrophy (SMA) and other diseases. The project, led by James Chambers, PhD, will evaluate access to…
BraunAbility has partnered with the nonprofit organization Laughing at My Nightmare (LAMN) to donate a wheelchair-accessible vehicle to a 6-year-old girl with spinal muscular atrophy (SMA). Fynlee Armstrong, a “sassy and spunky little girl” from Clyde, Texas, requires a wheelchair to move around. Over the last few years,…
Diagnostic testing company Ambry Genetics has launched a reproductive health program to increase access to carrier and prenatal screening for genetic disorders such as spinal muscular atrophy (SMA). The Comprehensive Assessment Risk and Education (CARE) program seeks to enhance the availability of carrier screening and noninvasive…
Development of an exoskeleton suit to help those living with progressive neuromuscular conditions that affect their upper-body movement will advance due to a £1.25 million (about $1.5 million) grant from the People’s Postcard Lottery in the United Kingdom. The “SMART Suit,” which targets people with disabilities such as…
Electric Bike Technologies (EBT) will donate 20 of its Liberty Trikes this year to help children with limited abilities get exercise and enjoy bike rides with their family and friends. In addition to helping young people with spinal muscular atrophy (SMA), the active-mobility tricycles, which can be folded,…
As part of ongoing efforts to have all newborns in the U.K. screened for spinal muscular atrophy (SMA), Muscular Dystrophy UK (MDUK) recently met with an ad hoc Scottish group to hear from parents and clinicians about life with SMA and the benefits of early diagnoses. The meeting, hosted…
