The purpose of SMA Europe is to provide a framework to stimulate collaboration and accelerate translational research pathways in SMA and promote patient care.
It was announced today that AveXis received FDA approval for Zolgensma®, the first gene therapy for paediatric patients with SMA. Zolgensma has been approved in the United States for the treatment of patients younger than 2 years of age who have been diagnosed with SMA and including those who were pre-symptomatic at diagnosis. This treatment […]
Novartis has sent the SMA community a letter announcing that enrollment for the ongoing clinical trial of branaplan is now closed. Branaplam is currently being assessed for safety and efficacy in a Phase 1/2 clinical study in Type 1 infantile-onset SMA. Novartis is pleased to announce that they have enrolled 25 infants into Part 2 […]
Roche presented data from two of its risdiplam trials, FIREFISH and SUNFISH, at the American Academy of Neurology annual conference. Risdiplam is an orally administered small molecule which encourages the “back-up gene”, SMA 2, to produce functional SMN protein. SMA Type 1 trial – FIREFISH FIREFISH is a trial of risdiplam on 21 infants with […]
Data from the NURTURE study presented at the American Academy of Neurology’s conference, showed that treatment of infantile-onset SMA (most likely to develop SMA Type 1 or 2) before the onset of symptoms resulted in motor milestone achievements more consistent with normal motor development. NURTURE is an open-label study of infants up to six weeks […]
Biogen has announced that data from a study on the safety and tolerability of Spinraza™ in individuals with later-onset SMA had been published in the peer-reviewed journal Neurology. Twenty eight patients, aged 5 to 19, who have, or are most likely to develop SMA type II or III were treated with Spinraza™ for over three […]