After bringing Zolgensma to market in 2019 as the first gene therapy for spinal muscular atrophy, Novartis is back with a ...

Delivery of onasemnogene abeparvovec into the intrathecal space was safe and effective for children with spinal muscular ...

Novartis AG (NYSE:NVS) announced safety and efficacy results from the Phase 3 program for investigational intrathecal ...

Novartis (NVS) announced positive safety and efficacy results from the Phase III program for investigational intrathecal onasemnogene ...

The SMA gene therapy candidate OAV101 IT, a version of Zolgensma, can be safely given to previously treated patients, per new ...

Scholar Rock , a late-stage biopharmaceutical company focused on advancing innovative treatments for neuromuscular diseases, cardiometabolic disorders, and other serious diseases where protein growth ...

A multidisciplinary approach, early diagnosis, and comprehensive caregiver education are crucial in effectively managing ...

Incorporating systems thinking into drug value assessment promises more realistic evaluations of the impact of new therapies, ...

What Are the Treatments for Spinal Stenosis? Spinal stenosis is a narrowing of the canal in your spinal column that affects mostly people age 50 and older. Nothing can cure it, but there are ...

What Is a Spinal MRI? A spinal MRI, or magnetic resonance imaging, uses powerful magnets, radio waves, and a computer to make clear, detailed pictures of your spine. The MRI may scan your whole ...

Patients with SMA, which has no cure, typically achieve a better quality of life through physical therapy, posture management, and, among other things, nutritional support. 4 Providing individualized ...

Therefore, option (c) is the correct answer. — A two-and-a-half-year-old girl has shown no signs of a genetic disorder — known as spinal muscular atrophy (SMA) — becoming the first person in the world ...