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

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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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Regenxbio receives FDA fast track designation for RGX-121 gene therapy for the treatment of MPS II

Regenxbio has announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for RGX-121. RGX-121 is a novel, one-time investigational treatment for Mucopolysaccharidosis Type II (MPS II), also known as Hunter syndrome, that is designed to deliver the

Awareness day 2018

MPS awareness in the news

You’ve been brilliant this MPS Awareness Day spreading the word about these diseases and paying homage to your MPS heroes. As well as a great social media campaign across Twitter, Facebook and Instagram we also reached new audiences through press

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New oral therapy for Fabry patients in development

Idorsia Pharmaceuticals, a Swiss biotech company, is developing lucerastat, a new oral therapy for patients with Fabry Disease. The Phase 3 clinical study MODIFY has just enrolled its first patients and will investigate the use of lucerastat monotherapy for the

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GSK signs strategic agreement to transfer rare disease gene therapy portfolio to Orchard Therapeutics

Agreement strengthens Orchard’s position as a global leader in gene therapy for rare diseases GSK takes 19.9% equity stake in Orchard and seat on board GSK and Orchard Therapeutics have announced a strategic agreement, under which GSK will transfer its