Tributes paid to determined twins
Loving tributes have been paid to the determination of twin brothers in North East Derbyshire who lived with Becker MD and who have died within 12 months of one another.
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Translarna MAA Q&A- an update
01/05/2019Find out more about the Translarna Managed Access Agreement
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Latest NICE update on Spinraza
30/04/2019NICE has released a statement that the discussions between NHS England and Biogen will be discussed at another committee meeting next week on Wednesday 8 May.
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Clinical trial for centronuclear myopathy given the green light in the UK
30/04/2019The drug company Dynacure has announced today that UK regulators have approved its UNITE-CNM phase I/II clinical trial.
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New activity study open to recruitment
30/04/2019To participate in the new study, you’ll simply need to live your life as usual and complete some questionnaires as well as wear an activity monitor for certain periods.
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Making strong friendships by volunteering for MDUK
29/04/2019"I wanted to do more. I have previously fundraised but I also wanted to help and to give something back, any way I can."
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Ex-Army dad set to take on the Great North Run 2019
29/04/2019“I decided to run for MDUK as I have a form of the condition myself. I thought it would make sense to run for a worthy charity that could eventually help me later on in life."
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Fulcrum repurpose drug for FSHD
24/04/2019Fulcrum Therapeutics has obtained rights to losmapimod from GSK. Fulcrum is interested in the drug because it has found that it can switch off DUX4 in FSHD cells.
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New stem cell therapy shows promise in Duchenne trial
23/04/2019Capricor Therapeutics has recently published the final results from its HOPE-Duchenne clinical trial. This study evaluated the safety and efficacy of a cell therapy called CAP-1002
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Positive preliminary results from SMA gene therapy trial
17/04/2019The trial results so far show that children treated with Zolgensma are living longer and achieving significant development milestones.
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2018 Winners describe the Newton Oxford Town and Gown 10k run
17/04/2019The history and scale of the Newton Oxford Town and Gown 10k run was a complete surprise to 25 year old Charlie Parnell, who was the first woman across the line last year.