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 >
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...
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...
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...
We are disappointed to hear that Abeona Therapeutics has decided to end enrollment for their clinical trial (Study ABT-003) for MPS IIIA children aged 2-18 years with a Developmental Quotient score less than 60. The company has recently conducted a review of...
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...
