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

Zolgensma is a prescription drug that’s used to treat spinal muscular atrophy (SMA) in young children. Zolgensma’s cost may depend on factors such as your child’s treatment plan and whether ...

Multidisciplinary coordination across prescribing teams, nursing, laboratory medicine, finance, and infusion centers is crucial for gene therapy delivery in Duchenne muscular dystrophy (DMD).

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

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

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 ...

Thiruvananthapuram: Health minister Veena George announced the launch of the Vishu Kaineettam project, aimed at supporting ...

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

The potential for cell and gene therapies is growing, offering groundbreaking treatments for rare genetic disorders and ...

Natco Pharma updates on Risdiplam launch in India, Delhi High Court denies Roche's injunction plea, pricing set at ₹15,900.

The parents of a boy who had an unapproved spring implanted in a spinal surgery without their consent or knowledge have said ...