Current Treatment Options for MPS I and Gene Therapy
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 >>
A Guided Pathway to the Clinical Trials Process
Courageous Parents Network has a Guided Pathway designed to present families with a clear-eyed picture of the clinical trials process, through the lens of families, researchers and professionals, and to empower you with information and perspective to help you...
What’s On Your Summer Reading List?
A list of books for children to teach them how everyone is unique how to turn feelings of sadness, anger and fear into happiness and how to find kindness and positives in each day.
Rare Revolution’s Magazine on Long-Term Caregiving
Rare Revolution’s online magazine. This edition recognises the unrelenting mental and physical strains of caregiving.
Mental Health Education Webinar Series
The Rare Disease Foundation has published five helpful videos on their YouTube channel from their recent Mental Health Webinar.
A Guide to Help Siblings Cope
Courageous Parent Network has published a guide Communicating Effectively and Compassionately to Help Siblings Cope.
Quebec Is the First In Canada to Develop a New Rare Disease Policy
The Canadian MPS Society welcomes the news that the Government of Quebec has unveiled its policy for rare diseases…
Promising News For Sanfilippo (MPS IIIA) Treatment
Lysogene has just provided promising data from their clinical trials for the efficacy of Gene Therapy LYS-SAF302 in the treatment of MPS IIIA.
What’s Morquio? By Titus Aged 3
Titus and his family have made a series of videos to show what it’s like to grow up with Morquio (MPS IV).
“Our Voices” Podcast: Saving Ryan
Saving Ryan is the inspiring story by physician-scientist Dr. Emil Kakkis about his journey to develop a new, first-ever treatment for MPS.