Listen to Bob and his family telling their story on BBC Three Counties Radio on Sunday 24 June.
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
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
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
individual and families
supported in 2015
rare disease supported
£ given research projects