As Christmas and the end of the year draw near, I’m reflecting on what 2021 has meant to me. If I look at the past year through a pessimistic lens, I could easily say, “Nothing has changed.” I’m still stuck at home because of the pandemic, and there’s still…
The team of researchers who discovered and developed Evrysdi (risdiplam) — the first at-home oral therapy for spinal muscular atrophy (SMA) — was selected as the winner of two prestigious treatment discovery awards. These are the Society for Medicines Research (SMR)’s 2021 Award for Drug Discovery and the British Pharmacological…
Scholar Rock announced the full design of the upcoming Phase 3 clinical trial testing apitegromab, its experimental muscle-directed therapy for spinal muscular atrophy, in children and young adults with types 2 and 3 SMA who are unable to walk. Apitegromab will be given as an add-on therapy to…
Let’s start this story about Evrysdi (risdiplam) from the beginning. About a year ago, the chronic neuropathy in my scalp that had been bothering me for three years worsened to a point of concern. It started as a mild and slightly nauseating throbbing from the back of my skull…
The National Institute for Health and Care Excellence (NICE) has recommended that Roche’s Evrysdi (risdiplam) be covered by England’s national health service (NHS) and provided at low or no cost to eligible spinal muscular atrophy (SMA) patients as part of a managed access agreement. The current recommendation and its…
In terms of “things I was expecting,” my mom coming down with COVID-19 was not one of them. The Albers family is notorious for doing too much at once. One of my caregivers often jokes that we’re the busiest people she knows. If we’re not renovating something, there’s a good…
Scholar Rock is on track to initiate a pivotal Phase 3 trial of apitegromab in non-ambulatory patients with type 2 and type 3 spinal muscular atrophy (SMA) by the end of 2021, according to a company press release. The planned randomized, double-blind, and placebo-controlled trial will focus on people…
The blood levels of neurofilaments — a marker of nerve cell damage — and the results of a nerve-muscle test called compound muscle action potential (CMAP) may serve as a biomarker of disease onset and treatment response in infants and children with spinal muscular atrophy (SMA) up to 3…
Motor function and stabilizing or improving breathing — along with oral administration — are the factors that adult patients and caregivers of children with spinal muscular atrophy (SMA) type 2 or 3 who cannot walk value most when deciding on available treatments, a U.K. survey study shows. Avoiding…
Roche and its subsidiary Genentech are launching a Phase 2/3 clinical trial to evaluate the safety and efficacy of GYM329 (RO7204239), an investigational anti-myostatin antibody, in combination with Evrysdi (risdiplam) in children with spinal muscular atrophy (SMA). The MANATEE trial has two parts. In part one, 36…