Mary Chapman,  features writer—

SMA News Today, having launched forums and added regular columnists, is now running a month-long series to coincide with Spinal Muscular Atrophy Awareness (SMA) Month that highlights the challenges and perseverance of patients and caregivers. Called “31 Days of SMA” and running through August, the series spotlights a person each…

More than 90% of rare diseases have no federally sanctioned treatment. So when news came of an approved new therapy for spinal muscular atrophy (SMA), Zolgensma, celebrations abounded. But as is often the case, questions soon followed: Where is the medicine available? How do we deal with insurance companies?…

The Canadian province of Saskatchewan has joined that of Quebec in granting access to Spinraza (nusinersen) therapy to a wider range of people with spinal muscular atrophy (SMA), Biogen Canada announced in a press release. Only those with type 1 SMA, the disease’s most severe form, had access to Spinraza.

While the Canadian Association of Drugs and Technologies in Health (CADTH) recommended that the spinal muscular atrophy treatment Spinraza should be made available to a broader population of patients, it said public funding should not cover patients older than 12 — a determination that the treatment’s developer Biogen called…

Spinraza (nusinersen) has been approved by China’s National Medical Product Administration (NMPA) for the treatment of the most common form of spinal muscular atrophy (SMA). Marketed by Biogen and increasingly available worldwide, Spinraza is the first approved treatment in China for SMA, a disease that results in progressively…

Citing the deteriorating health of a child with spinal muscular atrophy (SMA), a member of the British Parliament is pressing for the treatment Spinraza to be available in England. The medicine is under review by Britain’s independent, government-funded National Institute for Health and Care Excellence (NICE), which decides…

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…

Scores of events are afoot worldwide to mark Feb. 28, Rare Disease Day 2019. The activities aim to raise awareness about rare diseases and the millions of people — estimates run as high as 350 million — they are thought to impact. Across countries, patients, caregivers and advocates will paint faces, wear…

The U.S. Food and Drug Administration (FDA) is updating its 2015 draft guidelines for drug discovery in rare diseases, with new guidance on natural history — how disorders such as spinal muscle atrophy (SMA) run their course if untreated — the choice of “efficacy endpoints” in clinical…

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…