World Toilet Day 2020 aims to raise awareness about the 2.6 billion people worldwide who don’t have access to toiletsRead more
Chloe Ball-Hopkins, an ambassador for Muscular Dystrophy UK, speaks on the importance of Purple Tuesday for improving the customer experience for disabled people.
Trailblazers network moves over to charity Whizz-Kidz
NICE widens scope of its appraisal of Zolgensma TM
On Monday 17 August, Roche announced that the European Medicines Agency (EMA) has validated the Marketing Authorization Application (MAA) for risdiplam for patients with SMA.
The oral spinal muscular atrophy (SMA) drug Risdiplam has been approved by the FDA (U.S. Food and Drug Administration).
Two big announcements on Changing Places Awareness Day
More NHS trusts to offer Spinraza to adults
The SMA gene therapy Zolgensma has today been granted conditional approval by the European Commission (EC).
Read NICE statement about the impact of COVID-19 on access to Translarna.
Read NICE statement about the impact of COVID-19 on access to Spinraza.