Author: Andi Thornton
Cost of Living Grant
Yesterday Muscular Dystrophy UK launched a Cost of Living Grant for people living with muscle-wasting or weakening conditions.
ZOLGENSMA UPDATE
The pause in access to treatment with Zolgensma in children with type 1 SMA aged older than 12 completed months has been lifted.
ZOLGENSMA UPDATE
The pause in access to treatment with Zolgensma in children with type 1 SMA aged older than 12 completed months has been lifted.
Information about Apitegromab – The new treatment from Scholar rock
Recently the FDA designated Apitegromb for fast tracking as a treatment for SMA. We wanted to provide some additional information about this treatment, read on for more information and trial details.
New SMA Drug in phase 3 trials fast tracked
The US Food and Drug Administration (FDA) has granted Fast Track designation for Biohaven’s new anti-myostatin adnectin, taldefgrobep alfa, to treat spinal muscular atrophy (SMA).
Do you know about the Council for Disabled Children?
TreatSMA is signposting families to the Legal Handbook 3rd edition uploaded by the Council for Disabled Children.
UK SMA Patient Registry need your help
The UK SMA Patient Registry has asked TreatSMA to reach out to the community to help the research team at Columbia University, New York who are currently running a project focused on fatigue in SMA. The survey criteria is for eight years old and above and not restricted to any type of SMA or any country.…
New four year data for Roche’s Evrysdi reinforce long-term efficacy and safety profile
Data from pivotal SUNFISH study showed increases in motor function observed during the first year were maintained through the fourth year, while the overall rate of adverse events continued to decrease Data confirm long-term efficacy and safety profile of Evrysdi in a broad range of people with Type 2 and non‑ambulant Type 3 SMA More…