Aug 1, 2024

Dear Members,

The energy within our community has been incredibly positive, and I am continually inspired by the unwavering support and commitment of each of you. Together, we are making strides towards a brighter future for those affected by Rett Syndrome.

We have some promising news on the horizon. Health Canada is on track to approve the first drug for Rett Syndrome from Acadia Pharmaceutical by the end of this year. This milestone represents a significant step forward in our mission to improve the lives of those we support.

The successful reports emerging from Taysha Gene Therapies in Canada are particularly encouraging. As you may know, Canada was the first country to embrace a gene therapy program for adults, and we are thrilled that, Health Canada has approved Taysha’s gene therapy Clinical Trial Application for pediatrics commencing in Montreal, Canada under Dr. Rossignol’s leadership. This progress brings hope to our entire community.

Unfortunately, the recent Anavex trial involving our Canadian members did not yield statistically significant results. However, Anavex remains dedicated to our cause and will be reevaluating their approach. They plan to develop a new strategy and initiate additional trials. We remain hopeful that these efforts will lead to breakthroughs that will benefit our members.

ens opportunities for new individuals to join our board. If you have a passion for making a positive impact and a desire to contribute to our mission, we encourage you to consider applying for these positions.

At the Ontario Rett Syndrome Association, we remain committed and steadfast in our efforts to advance the quality of life for all those impacted by Rett Syndrome. Our mission is clear: to ensure that individuals with Rett Syndrome are enabled to achieve their full potential and enjoy the highest quality of life within their community.

Thank you for your continued support and dedication. Together, we will continue to make a difference.

Warm regards,

Sabrina Millson, President

Ontario Rett Syndrome Association

As O.R.S.A. continue to follow our strategic plan, we have highlighted the accomplished activities that align with our values and mission below.

Pillar #1

Increasing the Family Networking and Support

To provide clarity for the organization to empower and support caregivers and their loved ones with Rett syndrome.

Accomplishments:

• Hosted the “Stronger Together” parent and caregiver conference for families, professionals, and industry
• Provided real time support for families, clinicians, and sponsors
• Enhanced website content and security
• Ensured all members receive updates via email and social media
• Created an “Everyday Gift Ideas” package for families
• Supported the 2nd annual support for AAC Flash Camp
• Celebrated 10 years of the Canadian Rett Syndrome Registry

Pillar #2

Improving Health System Relations

By ensuring that our families are supported through the health system.

Accomplishments:

• Facilitated study requirements for ANAVEX2-73, A study in Pediatric Patients with Rett syndrome
• Assisted Taysha Gene Therapies in recruitment efforts and Health Canada approval for gene therapy programs in Canada
• Advocated for access of Daybue, the 1st commercially approved treatment for Rett syndrome by Acadia Pharmaceuticals
• Assisted Acadia with Health Canada priority review
• Provided Canada’s Drug Agency CADTH with an application to advocate for Daybue in Canada
• Engaged with other pharmaceutical companies regarding the opportunity for additional clinical trials in Canada
• Awarded Canadian researcher, Dr. Wright $50,000 dollars from the Hope Fund, 2024-2025 cycle
• Granted Canadian researchers Dr. Rossignol and Dr. LeRoux, $50.000 dollars from the Hope Fund, 2024-2025 cycle

Pillar #3

Create Awareness Strategies

To continuing to raise profile and visibility of O.R.S.A. nationally.

Accomplishments:

Collaborated with our Canadian registered associations, and several organizations outside of Canada (IRSF, RSAA)

• Coordinated the October awareness campaign #lightCANADApurple on October 29 including Proclamations within Ontario
• Ensured O.R.S.A. is more visible on social media
• Announced our annual O.R.S.A. artwork calendar
• Promoted Male Rett syndrome visibility and awareness content with links to research participation
• Attending the IRSF Ascend caregiver conference and scientist meeting

Pillar #4

Support Organizational Transition and Stability

By ensuring that the organization, support, and oversight of all O.R.S.A. activities and practices are in place and reviewed regularly.

Accomplishments:

• Conducted successful fundraising events, both signature events and 3rd party events
• Gained more sponsors to support O.R.S.A.’s mission
• Restructured committee members and vacant positions internally
• Completed review of O.R.S.A.’s by-laws, policies, and procedures
• Introduced code of conduct guidelines and policy for in-person and online discussions