This high dose nusinersen regimen for spinal muscular atrophy includes 50 mg loading doses and 28mg maintenance doses.

The singer, 34, revealed in January her now nine-month-old twins, Ocean and Story, had been diagnosed with SMA Type 1, a rare ...

Hindupur MLA Balakrishna supports fundraiser for toddler Keyansh, who needs ₹16 crore for vital treatment against Spinal Muscular Atrophy.

Jesy Nelson got emotional as she shared a sweet snap of her baby twins wearing leg splints on Wednesday - after celebrating a ...

The former Little Mix singer is on a career break as she focuses on her 10-month-old twins Ocean and Story and to campaign for testing at birth for SMA1 (Spinal Muscular Atrophy Type 1), a severe form ...

On March 31, 2026, Scholar Rock announced it had resubmitted its Biologics License Application to the FDA for apitegromab in spinal muscular atrophy, now incorporating both the Novo Nordisk-owned ...

Game of Thrones actor Michael Patrick died at 35 after battling motor neurone disease. His wife Naomi Sheehan shared an ...

Game of Thrones actor Michael Patrick's last social media post resurfaced after his death, drawing attention to a line where ...

Watchdog publishes protocol for a health technology assessment of the addition of congenital adrenal hyperplasia to the ‘heel prick’ test ...

When a biopharma company known for its expertise in a specific domain turns to a collaborator to expand its capabilities in ...

Many investors pay attention to mid-cap stocks because they have established business models and expansive market ...

It's time to ditch the one-size-fits-all approach in medicine. From cancer care and cardiology to TB and rare disorders, DNA ...