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News-Update
BBC Drama Best Interests information and support
We appreciate that watching this drama may lead to challenging emotions or raise questions or…
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Breaking News
NICE recommend new treatment option for late-onset Pompe disease
Pompe disease is a chronic, rare, genetic, progressive metabolic disease which results in severe…
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News-Update
New report published on newborn screening for rare conditions
Newborn babies in the UK are currently screened for nine – soon to be ten – rare, but serious,…
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News-Update
US FDA advisory committee votes on Sarepta SRP-9001 gene therapy to treat Duchenne muscular dystrophy ahead of a FDA’s final decision
UPDATE: On 24 May 2023, Roche published an update in which they stated that the FDA's final…
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News-Update
UK sites are now recruiting people with FSHD to take part in a clinical trial
We reported last year that Fulcrum Therapeutics announced that it will be starting a phase 3…
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Zolgensma Access Update
On January 9th 2023, following a small number of children having suspected significant adverse drug…
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NICE publishes new guidance for Zolgensma treatment in pre-symptomatic SMA
Zolgensma (Onasemnogene abeparvovec) is now recommended as an option for treating presymptomatic…
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News
Houses of Parliament
Muscular Dystrophy UK’s comment on the Chancellor’s Spring Budget statement
We were pleased to hear Rt Hon Jeremy Hunt MP confirm that the Energy Price Guarantee rise has been…
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Staff from Bidwells head office are all stood in front of the building with one arm in the, cheering. At the front is Matt Allen and Amy O'Hanlon shaking hands
Leading property consultant Bidwells announced as headline partner for historic Town and Gown 10k series
We are delighted to share the news that we have secured a headline partner for our historic Town…
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News
Changing places toilets
MDUK is delighted to share that over 100 new Changing Places toilets will be built across England following the allocation of the remaining funding from the Department of Levelling Up, Housing and Communities.
Following the allocation of the first round of funding last year, the remaining funds have now been…
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