Editor’s note: The SMA News Today team is providing in-depth coverage of the 2021 MDA Virtual Clinical and Scientific Conference, March 15–18. Go here to read the latest stories from the conference. Zolgensma — given alone, after, or in combination with another spinal muscular atrophy (SMA) disease-modifying therapy…
Editor’s note: The SMA News Today team is providing in-depth coverage of the 2021 MDA Virtual Clinical and Scientific Conference, March 15–18. Go here to read the latest stories from the conference. The Phase 2 RAINBOWFISH trial of Evrysdi (risdiplam) in newborns up to 6 weeks old with a…
Editor’s note: The SMA News Today team is providing in-depth coverage of the 2021 MDA Virtual Clinical and Scientific Conference, March 15–18. Go here to read the latest stories from the conference. Treatment with Evrysdi (risdiplam) is safe and increases the levels of SMN — the missing protein in spinal muscular…
PTC Therapeutics and the Spinal Muscle Atrophy (SMA) Association will collaborate in funding research into regenerative medicine, and into SMA in particular. With the advent of disease-modifying SMA therapies such as Evrysdi (risdiplam) and Spinraza (nusinersen), regenerative medicine — which aims to restore damage caused by…
It’s become a sort of ritual. Once every few weeks around 3 p.m., my mom will drop by my room. “So,” she’ll say, with ridiculous amounts of forced brightness. “So.” Code word for “We need to talk.” Code word for “I just checked the mail, and you’re not going to…
I gotta go climb some mountains, and I gotta do it now. That was the thought I focused on as I emerged from the pulmonary unit waiting room, weaving my wheelchair among sandstone fabric chairs arranged in a labyrinth of novice scale. Another six-month checkup appointment with pulmonary function…
Evrysdi (risdiplam) has been recommended for approval in the European Union (EU) to treat spinal muscular atrophy (SMA) patients, ages 2 months and older, with a clinical diagnosis of type 1, 2, or 3, or with one to four copies of the SMN2 “backup” gene. “Our close partnership…
Genentech’s Evrysdi (risdiplam) improved survival in infants with spinal muscular atrophy (SMA) type 1, and allowed them to achieve developmental milestones not usually seen in the natural course of the disease, according to one-year data from the dose-finding part of the FIREFISH trial. While the trial is still ongoing…
When Josylnn Jones McLaughlin moved to Guangzhou, China, to teach conversational English in 2012, she didn’t expect to bring home two girls, one with spinal muscular atrophy (SMA) and the other with spina bifida, when she returned to Anderson, South Carolina, more than a year later. She certainly…
Targeting a specific region of an intermediate molecule generated from the SMN2 gene increased the levels of functional SMN — the protein lacking in spinal muscular atrophy (SMA) — in cells derived from SMA patients, a study shows. In addition, combining this approach with a splicing modifier similar to…