Protalix BioTherapeutics’ pegunigalsidase alfa Receives Fast Track Designation from the U.S. Food and Drug Administration

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Protalix Biotherapeutics

Protalix BioTherapeutics’ pegunigalsidase alfa Receives Fast Track Designation from the U.S. Food and Drug Administration

Fast Track designation highlights high unmet medical need in the treatment of Fabry disease Protalix BioTherapeutics announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to pegunigalsidase alfa, or PRX-102, the Company’s plant cell-expressed recombinant,

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Important Announcement by the Society For Mucopolysaccharide Disease

As recently reported on the MPS Society website, Christine Lavery, our Group Chief Executive, sadly passed away on Tuesday 19th December 2017, in hospital, surrounded by her family following a brief illness. Christine tirelessly championed the MPS Society from its

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MPSII Clinical Trial Update – First potential treatment for children with Hunter disease and cognitive impairment misses its primary endpoints

The Society for Mucopolysaccharide Diseases (MPS Society) is disappointed to learn that Shire’s SHP609 clinical trial has missed its Primary Endpoints and failed to demonstrate Stabilization of Cognitive Decline in Children with Hunter Disease as stated in their press release

Pictures of some of the prizes available

National Draw winners

Thank you to everyone who bought and sold tickets for the National Draw in 2017. The money you raised from the draw will provide support for individuals and families affected by MPS and related diseases as well as funding research into treatments

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MPSIIIB Sanfilippo now included in Orchard Therapeutics and Manchester University collaborative programme

Orchard Therapeutics, a clinical-stage biotechnology company dedicated to transforming the lives of patients with rare disorders through innovative gene therapies, has announced that it has acquired an exclusive license to develop lentivirus-based autologous ex-vivo gene therapy for Sanfilippo syndrome type