The challenge of the obesity epidemic is widespread and one that even pediatricians face constantly,” a Connecticut doctor ...

Since 2016, the FDA has approved three disease-modifying treatments for spinal muscular atrophy, with several ...

After bringing Zolgensma to market in 2019 as the first gene therapy for spinal muscular atrophy, Novartis is back with an intrathecal formulation intended for older patients.

Taldefgrobep is a novel myostatin inhibitor that is optimized to block signaling of myostatin and other activin II receptor ligands, key regulators of muscle and fat metabolism. Biohaven is ...

We are looking for something similar or better than the C5 inhibitors that are currently on the market and approved for myasthenia gravis, which range from somewhere in the 1.5 to 2-point range.

That discovery launched an extensive effort in the pharmaceutical community to develop myostatin inhibitors to treat muscle diseases. Despite numerous drugs that were taken into clinical trials for a ...