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

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

With families scared 'to put a toe across the threshold of CHI', can the organisation get parents back on side ahead of the ...

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

The FDA has accepted for Priority Review the BLA for apitegromab for the improvement of motor function in patients with spinal muscular atrophy.

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 NIH is considering a catastrophic cut to indirect cost reimbursements for public universities, which would hurt the ...

Nearly six years ago, the Food and Drug Administration approved Zolgensma, a Novartis gene therapy for the fatal neuron-wasting disease spinal muscular atrophy. It heralded a new era of genetic ...

Girl from Bengal's Nadia suffers from spinal muscular atrophy Rs 1.4 crore still needed for girl's treatment which costs Rs 17 crore Suvendu Adhikari urges others to donate towards girl's treatment ...

Belfast boy Bobby Moore enjoyed an unforgettable experience on Sunday afternoon when he led Manchester United out for their ...