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

Nearly all children with spinal muscular atrophy who started on Evrysdi before symptom onset are walking after three years, ...

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

For people with spinal muscular atrophy, physical therapy can help support mobility, strength, and quality of life, regardless of SMA type.

SMA 特征性的呼吸系统病理生理改变主要为肋间肌无力、胸廓变形以及肺容量和通气能力降低，这些病理改变导致 SMA 患儿出现夜间和日间低通气、咳嗽减弱、痰液堵塞、反流与误吸、肺不张、反复呼吸道感染等呼吸系统问题[3]。

The parents of two boys born with a rare genetic disorder have demanded the rollout of a life-changing heel prick test – ...

With this judgment, Natco Pharma can now sell the Spinal Muscular Atrophy treatment drug for ₹15,900 compared to the previous ...

The Supreme Court upheld Delhi HC's interim order allowing Natco Pharma to manufacture and sell a generic version of Roche's ...

Roche has moved the Supreme Court challenging a Delhi HC order that allowed Natco Pharma to manufacture and sell a generic ...

Shares in US biopharma Scholar Rock closed 13% lower Monday after bad news on its spinal muscular atrophy (SMA) treatment ...

Roche sued Natco in early 2024 after learning that the Hyderabad-based company plans to launch a lower-cost generic version ...