Columnist Connie Chandler writes of a generational disconnect with new SMA patients, but says living fully must remain their ...

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 ...

Court upholds earlier order allowing cheaper SMA drug, calling affordable access more vital than Roche’s monopoly claim.

The division bench upheld the single-judge bench’s findings, which cited public interest in ensuring affordable access to SMA ...

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

CHENNAI: The Supreme Court of India on Friday dismissed an appeal filed by Swiss drugmaker F. Hoffmann-La Roche AG, which ...

Three-year-old Meher from Noida was diagnosed with Turner Syndrome at PGICH using QF-PCR, a first for the institute. Early ...

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

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]。

Kaitlin Batley, MD, a pediatric neurologist at Children’s Health and assistant professor at UT Southwestern Medical Center, discusses how physicians position different therapies for patients with ...