1-2 of 2 results for subject:"Muscular dystrophy"
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- subject:"Muscular dystrophy"
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- subject_t:"Muscular dystrophy" OR subject_t:"Duchenne muscular dystrophy" OR subject_t:"Myotonic dystrophy" OR subject_ses:11775
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To ask the Secretary of State for Health, what new treatments for Duchenne muscular dystrophy have been notified to the Horizon Scanning Research and Intelligence Centre in the last 12 months.
To ask the Secretary of State for Health, what new treatments for Duchenne muscular dystrophy have been notified to the Horizon Scanning Research and Intelligence Centre in the last 12 months.
In the last 12 months, the National Institute for Health Research Horizon Scanning Research and Intelligence Centre has identified and added to its database the following technologies for which Duchenne muscular dystrophy is an indication:
- ezutromid;
- FG-3019;
- follistatin;
- halofuginone hydrobromide;
- SRP-4045;
- SRP-4053; and
- vamorolone.
To ask the Secretary of State for Health, how many children are born each year in England with Duchenne muscular dystrophy; how many people are living with this condition; and how many such people do receive Translarna.
To ask the Secretary of State for Health, how many children are born each year in England with Duchenne muscular dystrophy; how many people are living with this condition; and how many such people do receive Translarna.
Information on how many children are born each year in England with Duchenne muscular dystrophy is not collected. However, it is estimated that there are around 2,500 people living with Duchenne muscular dystrophy in the United Kingdom.
Following the recommendation made by the National Institute for Health and Care Excellence in April 2016 and the announcement made by NHS England on 7 July, Translarna is now routinely commissioned for those individuals who have Duchenne muscular dystrophy and meet the relevant clinical criteria. Currently, there are approximately 50 patients receiving Translarna.