EVERYBODY GRIEVES: Webinar Recording
Grief is considered a "taboo" topic and should be avoided in conversation. As a result, even fewer people know how to respond to and process grief – both their own and that of others. Grief can be experienced not only after someone’s passing but also can be...
Run for Raza
On September 17, 2022, a group of my family and close friends were kind enough to come together to run the Tough Mudder Obstacle Race in memory of Raza Amin and the MPS Society. Tough Mudder is a team building endurance event series in which participants attempt...
Why is it imperative to recognise MPS early?
Kim Angel, Executive Director, Canadian MPS Society explains why recognising MPS early can save a child’s life.
The Rare Sibling Experience: Sibshops Support
Listen to a talk about the sibling perspective and the support available with Emily Holl, the Director of the Sibling Support Project. Emily is a social worker, writer, trainer, and sibling. Over the past 16 years, she has provided workshops, training, and groups for...
Results Of the Consultation On the PMPRB Price Review
On June 30, 2022, The Patented Medicine Prices Review Board (PMPRB) issued a Notice and Comment on its proposed approach for reviewing the prices of patented medicines during the period between the coming into force of the recently amended...
Anaesthesia Recommendations For Rare Diseases
Want to find out more about anaesthesia and how it relates to rare diseases? OrphanAnesthesia publishes anaesthesia recommendations for rare diseases in order to facilitate the work of anaesthesiologists and to improve patients’ safety. This a project of the...
MPS II is added to New Born Screening in the USA
The Department of Health and Human Services (DHHS) in the United States has approved adding MPS II as a condition to the recommended uniform screening panel (RUSP) for newborns. This enables several states to begin testing newborns immediately. Early diagnosis...
Ultragenyx Takes Over Gene Therapy For Sanfilippo Syndrome Type A (MPS IIIA)
In our March edition we reported that Abeona Therapeutics had decided to end enrollment for their clinical trial for MPS IIIA children. We are now happy to share that Ultragenyx will be taking over the clinical trials for AAV gene therapy ABO-102 (now UX111). “Based...
Another Potential Gene Therapy to Combat Against Hunter Syndrome MPS II
We are excited to share the news that REGENXBIO Inc. has announced its intention to file a Biologics License Application (BLA) in 2024 using the FDA's accelerated approval pathway for the treatment of Hunter Syndrome, Mucopolysaccharidosis Type II (MPS II). The...
Gene Therapy For Inherited Metabolic Diseases Explained
Ever wondered… How does gene therapy work?Why does it work better for some conditions that others?What is the difference between in vivo and ex vivo gene therapy?What are the ways of receiving gene therapy?How safe is it and what are the dilemmas surrounding gene...