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 [...]
MPS I Diagnostic Journey Animated Video
https://youtu.be/jSq5tQD4nxE
Symptoms of MPS I – Animated Video
Click to watch the video describing early symptoms of MPS I. https://youtu.be/h111oEHS7-c
MPS I Parents talk about their diagnostic journey
Click to watch the video of Canadian MPS I parents where they share their stories about the diagnostic journey of their children. https://youtu.be/tKflBtR6iYI
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 [...]
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 [...]