Despite significant advancements in spinal muscular atrophy (SMA) management, unmet needs persist, especially for patients ...

FDA-approved treatments for spinal muscular atrophy differ in their mechanisms of action, safety profiles, and administration ...

Funding medical research is about more than just numbers. It's real people,” says Ryan Manriquez, president of the UC ...

Imagine the joy of holding your newborn for the first time, only to hear doctors say your baby would live to the age of two.

Affordable generic version of SMA drug Risdiplam soon available in India, offering hope to patients and activists.

Intrathecal treatment with a vector-based gene therapy was associated with a greater improvement in motor function at 52 weeks compared with sham in patients aged 2 to 18 years with spinal muscular ...

The Supreme Court will review the Rs 50 lakh cap on the central government's aid for treating rare diseases like SMA. The ...

Can the medicine for treatment of rare disorder spinal muscular atrophy (SMA) be made available at lower price in India if it ...

The bench said pharma firm M/s F Hoffmann-La Roche Ltd, which manufactures the SMA drug Risdiplam priced at Rs 6.2 lakh a ...

It's that time of year when you can become a Hero for Hope and donate to ABC15's annual Telethon for Phoenix Children's ...

National Policy for Rare Diseases 2021 addresses diagnosis and treatment, categorizing diseases and advocating for affordable ...

Supreme Court examines pleas challenging 50 lakh cap on Centre's assistance for treating rare diseases like SMA.