Latest News

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Bob Stevens and family on BBC Three Counties Radio

Listen to Bob and his family telling their story on BBC Three Counties Radio on Sunday 24 June.

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Regenxbio receives FDA fast track designation for RGX-111 gene therapy for the treatment of MPS I

Regenxbio have announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for RGX-111. RGX-111 is a novel, one-time investigational treatment for Mucopolysaccharidosis Type I (MPS I), that is designed to deliver the human iduronidase (IDUA) gene directly

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Clinical trial for genome editing treatment in MPS I and MPS II announced

Sangamo Announces UK Authorisation Of Clinical Trials Evaluating Zinc Finger Nuclease In Vivo Genome Editing Treatments SB-318 For MPS I And SB-913 For MPS II Please note the clinical trial for the UK is expected to start towards the end

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Outcomes of consultation document leans towards a NO for treatment of Alpha Mannosidosis

NICE, the drug decision making body, publicly released their interim decision on the 23 May 2018 not to recommend velmanase alfa, the treatment for individuals aged 6 years and over suffering from Alpha Mannosidosis. Members of the public have until

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MPS in numbers

896

individual and families
supported in 2015

25

rare disease supported

3,364,628

£ given research projects
between 1984-2010