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Paul Hastings' journey from patient to chair of the Biotechnology Innovation Organization bizjournals.com - get the latest breaking news, showbiz & celebrity photos, sport news & rumours, viral videos and top stories from bizjournals.com Daily Mail and Mail on Sunday newspapers.
Vor Biopharma Names Matthew R. Patterson as Chairman and Promotes Tania Philipp streetinsider.com - get the latest breaking news, showbiz & celebrity photos, sport news & rumours, viral videos and top stories from streetinsider.com Daily Mail and Mail on Sunday newspapers.
Amicus Pompe disease therapy joins early access scheme 9th June 2021 Patients with late-stage Pompe disease who are failing to respond to enzyme replacement therapy will now have early access to an alternative treatment option, after Amicus Therapeutics cipaglucosidase alfa with miglustat was approved for the UK s Early Access to Medicine Scheme (EAMS). The Medicines and Healthcare regulatory Products Agency (MHRA) has issued a positive scientific opinion for use of the treatment in adults with late-onset Pompe disease previously treated with alglucosidase alfa, based on currently available data. This means that doctors will be able to prescribe the treatment to eligible patients with the rare, inherited disorder while its licensing is being considered by regulators. ....
Epidermolysis Bullosa benzinga.com - get the latest breaking news, showbiz & celebrity photos, sport news & rumours, viral videos and top stories from benzinga.com Daily Mail and Mail on Sunday newspapers.