Gene therapy is linked to better nonmotor outcomes in spinal muscular atrophy type 1 compared to first-line nusinersen, new ...

Imagine a child born in a family with a rare disease, whose treatment would cost more than 17 crore for a single dose. The ...

Please provide your email address to receive an email when new articles are posted on . At the time of last follow up, 82.6% of children born with SMA type 1 from 2016 on were still alive. Of the 140 ...

The Chinese report details the case of a 6-year-old female patient with type 1 SMA who was successfully weaned off prolonged invasive ventilation after beginning treatment with nusinersen. A late ...

Researchers compiled data from studies of oral Evrysdi (risdiplam), Spinraza (nusinersen), and Zolgensma (onasemnogene abeparvovec), finding evidence that risdiplam may be a favorable alternative to ...

(RTTNews) - Novartis (NVS) announced new data that reinforce the transformational benefit of Zolgensma (onasemnogene abeparvovec), an essential one-time treatment for spinal muscular atrophy or SMA.

– Infants treated with Evrysdi maintained or continued to improve in measures of motor function, including their ability to sit without support for 5 and 30 seconds – – Evrysdi has proven efficacy in ...

ALLENDALE, Mich — A family in the Allendale area received two rounds of unexpected news, after both of their kids were diagnosed with Spinal Muscular Atrophy. February 28 marks Rare Disease Day, and ...

Some patients with later-onset spinal muscular atrophy (SMA) type 2 and type 3 had improved motor function when the investigational monoclonal antibody apitegromab was added to their treatment, the ...