This Is A Custom Widget

This Sliding Bar can be switched on or off in theme options, and can take any widget you throw at it or even fill it with your custom HTML Code. Its perfect for grabbing the attention of your viewers. Choose between 1, 2, 3 or 4 columns, set the background color, widget divider color, activate transparency, a top border or fully disable it on desktop and mobile.

This Is A Custom Widget

This Sliding Bar can be switched on or off in theme options, and can take any widget you throw at it or even fill it with your custom HTML Code. Its perfect for grabbing the attention of your viewers. Choose between 1, 2, 3 or 4 columns, set the background color, widget divider color, activate transparency, a top border or fully disable it on desktop and mobile.

Everything Else MPS

/Everything Else MPS

REGENXBIO Announces Positive Interim Data and Update for PHASE I/II Trial of RGX-121 for the Treatment of MPS II

REGENXBIO  announced interim data from Cohorts 1 and 2 of the ongoing Phase I/II trial of RGX-121 for the treatment of patients up to 5 years old diagnosed with Mucopolysaccharidosis Type II (MPS II). In addition, REGENXBIO announced plans to evaluate a higher dose of RGX-121 in a third cohort of patients at an increased dose of 2.0x1011 [...]

Abeona Therapeutics Announces Issuance of U.S. Patent for AIM™ Capsids

NEW YORK and CLEVELAND, Jan. 15, 2020 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (Nasdaq: ABEO), a fully-integrated leader in gene and cell therapy, today announced that the United States Patent and Trademark Office (USPTO) issued a patent covering next-generation adeno-associated virus (AAV) capsids generated using the University of North Carolina’s (UNC) AIMTM vector platform and that [...]

Hunter Syndrome “Listening Session” Feb 4, 2020

Dear Members, We are writing to invite you to participate in an important phone call to help inform staff of the U.S. Food and Drug Administration (FDA) about the patient and caregiver experience of living with Hunter Syndrome. This is part of a “Listening Session” being co-hosted by FDA’s Patient Affairs Staff (PAS) and the National [...]

Invitation to Participate in Research Study for Canadian Patients and Families with Lysosomal Storage Diseases (LSDs)

Invitation to Participate in Research Study for Canadian Patients and Families with Lysosomal Storage Diseases (LSDs)  This study is part of the PhD Program at the Carleton University Study Title: Access to Orphan Drugs in Canada: Integrating Patient and Policy Perspectives (Study #110105).   Dear patients and families with Lysosomal Storage Diseases (LSDs): We are conducting interviews [...]

Sangamo Therapeutics announced an update on its in vivo genome editing programs: SB-913 (mucopolysaccharidosis type II, or MPS II), SB-318 (MPS I)

    Message from Christeen Moburg, Sr. Director, Patient Advocacy, Sangamo Therapeutics MPS I & MPS II We have been encouraged and heartened to see preliminary evidence of successful in vivo genome editing in our MPS I and MPSII clinical studies. However, we have determined that no additional patients will be treated at this time with first generation [...]

Request for Proposals – Summer Studentship Research Grants

The Canadian MPS Society is once again offering a limited number of Summer Studentship Research Grants for medical students and university undergraduates. It is our hope that these opportunities may result in more individuals being attracted to research careers involving lysosomal storage disorders. The studentships are tenable at any university centre in Canada, and there is [...]