With about 100 spinal muscular atrophy (SMA) patients already treated with Zolgensma (onasemnogene abeparvovec-xioi) since the gene therapy’s May approval in the United States, and expected growth in newborn screening, the groundbreaking treatment that is also the world’s most expensive is off to a robust start, Novartis…
Next month’s annual conference of the National Organization for Rare Disorders (NORD) in Washington, D.C., couldn’t come at a better time, says Marshall Summar, MD, chairman of NORD’s board of directors. “The pace of discovery in rare diseases has gone from brisk to hypersonic,” Summar told Bionews Services, publisher…
A new and faster genetic test for spinal muscular atrophy (SMA), called SMA STAT, will be offered free of charge in the U.S. as part of the SMA Identified program. According to Biogen and Invitae Corporation, SMA STAT reduces the time for a definitive SMA diagnosis — through…
Online forums have markedly altered how patients, caregivers, and healthcare professionals communicate and connect. Even when miles apart, individuals can establish supportive relationships, share experiences and information, and sometimes just vent. Bionews Services, a leading online health, science, and research publication company, has been rolling out its…
Skye, a nine-month-old baby with spinal muscular atrophy (SMA) type 1, is reaching new milestones every week and is “the happiest child” after being treated with Zolgensma, according to her mother Kasia. “She is almost sitting, her head and neck are really strong, and she is reaching above…
Oklahoma suffers more tornadoes than any other state, has the highest per-capita rate of women in U.S. prisons, ranks second in the number of teen births per 100,000 teenage girls, and has the nation’s third-highest rate of uninsured residents — with 13.9% of all Oklahomans lacking health coverage. As if…
Like most families broadsided with an unexpected, rare diagnosis, my husband and I had never heard of the one attached to Jeffrey, our third baby. Spinal muscular atrophy (SMA) was completely foreign to us, so it came as another shock to learn that SMA was the leading…
A renowned Belgian neurologist and spinal muscular atrophy (SMA) expert has become a hero in Poland for helping dozens of Polish patients obtain early access to Biogen’s Spinraza (nusinersen), the first approved treatment for the disease. Poland’s SMA community says Laurent Servais never turns a patient…
[Editor’s Note: This is the first of a two-part series on the Lackey family of Arizona, whose daughter Stella became the first baby west of Missouri to be treated outside of a clinical trial with Zolgensma on July 3. Here, the family talks about how their daughter came to be…
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?…