Dr John Bach is a world renowned expert in non-invasive respiratory care. A legend in his field he has well over 600 papers published as well as books that I used across the world to help manage all types of…
Have you ever wondered what worms and SMA have in common? No, me neither, until now. Our very own science geek G hosted a webinar with Dr Maria Dimitriadi to talk about that and many other fascinating topics. Another fascinating…
We were delighted and truly honoured to have Baroness Tanni Grey-Thompson join us as part of SMA Awareness Month to talk about her life, her experiences, discrimination and answer some of your questions. Such a thought-provoking and and inspirational discussion…
We joined a session with Roche earlier this week to ask them questions following their recent announcement about risdiplam. The following FAQs have been checked for accuracy with Roche where they relate specifically to risdiplam.
Questions and answers apply to people living in England, Scotland, Northern Ireland and Wales.
A really interesting webinar hosted by our very own Joseph talking to Dan from the MHRA about the regulatory process associated with rare diseases and their involvement in assessing suitability and helping licensing drugs.
European Medicines Agency (EMA) has validated the Marketing Authorization Application (MAA) for risdiplam for the proposed use in patients with spinal muscular atrophy (SMA).
Mary has become an expert and the go to person in the US with regards to the amino acid diet, something Mary used on her own daughter at the age of 5, and attributes the stability to of her condition…
We were fortunate enough to have one of the U.K.'s leading consultants in Spinal Muscular Atrophy to spend some time with us and the community to talk about SMA treatments as well as answer some questions.