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Promising News For Sanfilippo (MPS IIIA) Treatment

Promising News For Sanfilippo (MPS IIIA) Treatment

by adminmps | Jun 17, 2022 | Clinical Trials, News, Research

Lysogene has just provided promising data from their clinical trials for the efficacy of Gene Therapy LYS-SAF302 in the treatment of MPS IIIA. Read Press Release >
Discontinued TAK-609 Clinical Trial for MPS II

Discontinued TAK-609 Clinical Trial for MPS II

by adminmps | May 26, 2022 | Clinical Trials, News, Research

The Canadian MPS Society met directly with Takeda Pharmaceuticals on May 11th, when Takeda shared with us that they will discontinue development of TAK-609, investigational intrathecal enzyme replacement therapy for MPS II, Hunter syndrome. Patients with MPS II...
NICE Recommends Vimizim For Treating MPS IVA Morquio

NICE Recommends Vimizim For Treating MPS IVA Morquio

by adminmps | Apr 29, 2022 | Clinical Trials, News, Research, Treatment

We are excited to hear that Elosulfase alfa – branded as Vimizin and made by BioMarin – has been recommended by NICE (National Institute for Health and Care Excellence) for treating MPS IVA Morquio in England and Wales. Newly diagnosed patients and...
Recruiting for Clinical Trial: MPS-II (Hunter Syndrome) with Pabinafusp-Alfa

Recruiting for Clinical Trial: MPS-II (Hunter Syndrome) with Pabinafusp-Alfa

by adminmps | Apr 29, 2022 | Clinical Trials, News, Research

Mathias Schmidt, PhD, President and CEO of JCR Pharmaceuticals USA, discusses long-term efficacy and safety data of pabinafusp-alfa (Izcargo) in mucopolysaccharidosis type II (MPS II; Hunter syndrome). (Data from this study was recently presented...
Rapid Test Expected to Help in Developing Treatments for Sanfilippo

Rapid Test Expected to Help in Developing Treatments for Sanfilippo

by adminmps | Apr 29, 2022 | News, Research

A new study shows a 30-minute, inexpensive, and easy-to-use test may help measure the activity of NDST1, one of the main enzymes involved in heparan sulfate, the sugar molecule that build ups and causes Sanfilippo syndrome. Sanfilippo syndrome, or MPS III, is a...
Research Highlights from the 2022 WORLD Symposium:

Research Highlights from the 2022 WORLD Symposium:

by adminmps | Mar 4, 2022 | Clinical Trials, News, Research

MPS I (Hurler syndrome) Orchard Therapeutics Announces Interim Data for OTL-203 Showing Positive Clinical Results in Multiple Disease Manifestations of Mucopolysaccharidosis Type I Hurler Syndrome (MPS-IH). Read the full report > SiglionTherapeutics, Development of a...
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