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2024年12月18日 · DMTs have transformed SMA type 1 in Italy, nationwide study finds. Nearly 4 of every 5 infants with spinal muscular atrophy (SMA) type 1 born in Italy during the era of disease-modifying therapies (DMTs) are still alive, illustrating that such treatment has “radically changed the course of … type I SMA,” with “much higher survival,” according to a nationwide…
Top 10 SMA stories of 2023 - SMA News Today
2024年1月3日 · No. 9 – New gene therapy shows promise in SMA mouse model. In May, CANbridge Pharmaceuticals announced that its investigational second-generation gene therapy, called CAN203, led to greater motor function improvements and longer survival in a mouse model of SMA compared to a benchmark treatment reported to be similar to Novartis’ approved gene therapy Zolgensma (onasemnogene abeparvovec-xioi).
About Us - SMA News Today
SMA News Today is a digital platform intended to provide the spinal muscular atrophy (SMA) community with the most recent news and information on SMA, as well as first-hand community perspectives from our patient and caregiver columnists.. All articles on SMA News Today are original content produced by in-house writers and editors. Occasionally, we seek the opinion of outside experts for ...
1 in 59 women in region of China found to be SMA carrier: Study
2024年2月16日 · Hospital launches 3-stage SMA carrier screening for women. SMA is a rare genetic condition usually caused by inherited mutations in the SMN1 gene that lead to the progressive loss of motor neurons, the specialized nerve cells that control voluntary movements. This ultimately results in the disease symptoms, such as muscle weakness and atrophy, or difficulties breathing, speaking, and swallowing.
Top 10 SMA Stories of 2019 - smanewstoday.com
2020年1月3日 · In September, Biogen announced its plan to initiate a global Phase 2/3 clinical trial (NCT04089566), called DEVOTE, to evaluate whether higher doses of Spinraza promote greater motor improvements in up to 126 people with infantile- or later-onset SMA.DEVOTE will also assess the safety, tolerability, and pharmacokinetics (uptake, distribution, and elimination in the body) of those higher doses ...
About Us – HCP SMA News Today
SMA News Today is a digital platform intended to provide the spinal muscular atrophy (SMA) community with the most recent news and information on SMA, as well as first-hand community perspectives from our patient and caregiver columnists.. All articles on SMA News Today are original content produced by in-house writers and editors. Occasionally, we seek the opinion of outside experts for ...
Faces of SMA – SMA News Today
Throughout August, SMA News Today is recognizing Spinal Muscular Atrophy (SMA) Awareness Month with a multimedia celebration featuring a collection of written essays and video interviews from SMA patients and caregivers. These stories highlight some of the unique challenges of living with SMA, including a diverse range of topics: relationships, parenthood, careers, …
HCP SMA News Today – The Web's Leading Resource For People …
Possible solution found to SMA patient’s discomfort in wheelchair. One of my patients, Michael, who has spinal muscular atrophy, has continued to experience increased pain and pressure along his coccyx and into his right hip while sitting in his wheelchair all day.
Top 10 SMA Stories of 2022 - SMA News Today
2022年12月29日 · No. 6 — “#MDA2022 – ‘Amazing’ Walking Ability Seen in SMA Children on Spinraza” The Phase 2 NURTURE trial (NCT02386553) is testing the injectable therapy Spinraza in 25 children genetically diagnosed with SMA, who started treatment before the age of 6 weeks, prior to showing any symptoms. At the Muscular Dystrophy Association annual meeting, researchers presented the trial’s ...
New large-scale SMA carrier screening test most effective: Study
2024年10月14日 · People who inherit one copy of the mutated gene and one healthy copy are carriers. While they won’t develop SMA or disease symptoms, they can pass the condition on to their children.. Cells have a second, almost identical SMN2 gene that can compensate, but only partially, for the loss of SMN1.The number of SMN2 copies, which naturally varies across populations, generally influences SMA ...