Onasemnogene abeparvovec is an AAV-based gene replacement therapy that delivers the SMN1 gene into the anterior horn cells of the spinal cord. Of note, at seven days prior to administration of the ...

Sales growth driven by continued strong performance from Entresto (+31% cc), Cosentyx (+25% cc), Kesimpta (+49% cc), Kisqali (+49% cc), Pluvicto (+42% cc) and Leqvio (+114% cc) Core operating income ...

Background: Spinal and bulbar muscular atrophy (SBMA) or Kennedy disease [OMIM ... consent form and allowed anonymised publication of their clinical information and photos. The study included all ...

The 18-month-old, suffering from a rare genetic disease called Spinal Muscular Atrophy (SMA ... the scalpel after a gap of two years. Dr HD Ranganath, Congress MLA from Kunigal ...

In a LinkedIn post, Monisha shared the challenges of travelling with her 14-year-old daughter, Keya, who has Spinal Muscular Atrophy (SMA). Understanding the early signs, potential complications ...

Three patients with spinal muscular atrophy had improved muscle strength and could walk farther after a month of daily spinal ...

A Boston-area biopharmaceutical firm has submitted a biologics license application to the FDA for its investigational, fully ...

who are living with Spinal Muscular Atrophy (SMA) with the support of her mother, sister and teacher. Ms. Keya was felicitated by the Visakha Rasagna Vedika, a cultural organisation, at the Public ...

Seeking urgent Union government intervention to overcome the lack of affordable access to medicines for treating Spinal Muscular Atrophy (SMA) and the need to allow local manufacturers to make ...

So the outcome and the long-term outlook for our patients with SMA, particularly in the adult populations are that they are now stronger and living longer. And so as a result of that, they're able ...