Alexion MPSIIIB clinical trial update


MPS Society logo

Alexion MPSIIIB clinical trial update

Monday 17 July 2017 Alexion MPSIIIB Enzyme Replacement Clinical Trial Dr Suresh Vijay, Clinical Trial Principal Investigator at Birmingham Children’s Hospital has confirmed that it is business as usual until he gets clarification from Alexion on the status of the

MPS Society logo

Clinical trials for MPSIIIB stopped

14 July 2017 The MPS Society has just been informed by the PI Dr Suresh Vijay from Birmingham Children’s Hospital that the NGLU clinical trials for MPSIIIB have been stopped with immediate effect. At this time neither the CRO, PPD,


Who do you know who has shown courage over adversity?

MPS Society anniversary awards May 2017 is the 35th Anniversary of the founding of the Society for Mucopolysaccharide Diseases. To celebrate this milestone the Board of Trustees will at the MPS Gala Dinner at the conference on Saturday 8 July

Ultragenyx announces investigational therapy for MPS VII accepted for review

Ultragenyx Announces Recombinant Human Beta-Glucuronidase Biologics License Application and Marketing Authorization Application Filed and Accepted for Review; FDA Grants Priority Review Status Ultragenyx Pharmaceutical Inc., a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases,

Abeona Therapeutics logo

Abeona Therapeutics Receives Regulatory Approval to Initiate Clinical Trial in Australia with ABO-102 Gene Therapy For Patients with MPS IIIA

Abeona Therapeutics Inc., a leading clinical-stage biopharmaceutical company focused on developing novel gene therapies for life-threatening rare diseases, has announced Australian regulatory approval to initiate a Phase 1/2 for the ABO-102 gene therapy program for patients with Sanfilippo syndrome type