With each new advance in medicine comes ethical dilemmas, from fertility treatments and newborn screening, to vaccinations, gene therapies and euthanasia. But rare diseases and the expensive therapies needed to treat them — particularly in an age of scarce economic resources — almost always entail “tragic choices,” warned Avraham Steinberg,…

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…

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…

Danyelle Sun, a Milwaukee mother of two children with spinal muscular atrophy (SMA), says Biogen’s Spinraza (nusinersen) has rescued both her kids from potentially lifelong disabilities. Sun, 35, is the Wisconsin state ambassador for NORD, the National Organization for Rare Diseases. Her 7-year-old daughter, Ruby, has SMA type 3,…

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,”…

For the first time ever, British spinal muscular atrophy (SMA) patients and their families have taken to the streets to voice anger over a draft recommendation not to provide government funds to cover the cost of Spinraza (nusinersen) to treat the disease. Apparently, the noisy protests have had…

Canada’s healthcare system is excellent for people with common ailments like diabetes or high blood pressure, but it’s “basically failing the nearly three million Canadians with rare diseases.” So says Durhane Wong-Rieger, president and CEO of the Canadian Organization for Rare Disorders (CORD), a Toronto-based network representing 102 patient advocacy…

A program offered through a partnership between Invitae Corporation and Biogen offers free genetic testing to infants, children and others with clinically diagnosed spinal muscular atrophy (SMA) or suspected of having the disease. The program, SMA Identified, aims to increase access to genetic testing for the SMA community, so…

Cure SMA will host the world’s largest regular gathering on spinal muscular atrophy next month in Dallas, bringing together leading SMA researchers and clinicians, as well as families living with the disease. The 2018 Annual Cure SMA Conference is set for June 14-17 at the Hilton Anatole Hotel. Its first three days…

Type 1 spinal muscular atrophy (SMA) babies treated with AveXis’ gene therapy candidate AVXS-101 continue to achieve unprecedented motor milestones and do not require breathing or nutritional support, according to recent data from the company. AveXis presented updated results from Phase 3 and Phase 1 trials…