The problem with disability pride is that it’s a little bit mythical. It’s not something you can describe or explain. It’s an enigma. A paradox. It shouldn’t exist, but it does, and the illogic of it — valuing something the world abhors — makes the concept seem impossible. You…

Novartis is stopping the clinical development of branaplam (LMI070), its experimental oral treatment for spinal muscular atrophy (SMA) being evaluated in a Phase 1/2 clinical trial. According to the company’s announcement, this “difficult” decision was based on the “rapid advancements in the SMA treatment landscape in recent years,”…

A series of toxicology studies in rats and macaque monkeys confirmed the safety and tolerability of apitegromab, an investigational spinal muscular atrophy (SMA) therapy, supporting its ongoing assessment in patient trials, a study reported. The study, “Preclinical Safety Assessment and Toxicokinetics of Apitegromab, an Antibody Targeting…

I can still see the golden afternoon of my first tea party like it was only yesterday. I swung open the wooden cottage gate. In the center of a secret garden surrounded by dense evergreen hedgerows, a buffet table sat beneath the spotlight of the sun, awaiting guests. An elegant…

SadBaby, an organization that raises donations to support children with spinal muscular atrophy (SMA), is launching a crowdfunding platform. The project, based on digital currency donations, intends to gather funds from supporters and investors to help families financially with the costly treatments associated with SMA. “SadBaby is doing…