In this radio interview, Dr. Wang discusses the current treatment options for MPS I as well as the work he is doing to assess the safety and efficacy of gene therapy (RGX-111) for this rare disease. Listen to the interview >>
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...
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...
