Many thank to the International Rett Syndrome Foundation @rettsyndrome.org for hosting the 2024 ASCEND family conference. It was wonderful and heartwarming to re-connect with families, friends, health care professionals and Industry. We are excited to announce our continued partnership with the IRSF, industry and new venders that can help our community.
Community Update
IRSF message “Change is required, courage is needed, community is the fuel, hope is mandatory” There are currently over 20 companies working hard on different drugs and therapies with the ultimate goal to cure Rett syndrome. Many new tools are available to support families on their website. Information provided from this conference will be available to families.
Acadia Pharmaceutical Update
The Canadian Pharma team is on track for Health Canada approval this fall. It’s exciting to know that the 1st drug approved for Rett could be available to Canadian’s as early as 2025.
O.R.S.A. is in the process of submitting an application to the Canadian Agency for Drug and Technologies in Health (CADTH) advocating on behalf of all the Rett associations in Canada and families to allow access to DayBue to Canadians.
Coming soon, education will be provided to medical professionals, families and caregivers. We suggested a tip sheet of ‘lived experience’ from families already on Daybue to reduce the likelihood of side effects. We will continue to ask for tools to help make this a smooth transition.
Taysha Gene Therapies Update
Our partnership with Taysha has been very successful and continues to be positive and fruitful as we paved the way for the 1st gene therapy trial to be available to adults in Montreal, Canada. Many thanks to Dr. Rossignol and team for trailblazing this potential treatment!
Anavex Life Sciences Update
Anavex appreciates all the Canadian families taking part in their clinical trials. As this trial comes to an end, they have informed us that other drugs trials are coming down the pipeline.
Neurogene Update
16 participants will be enrolled in total from the following sites, US, UK, and AUS. More information about this trial can be found on clinicaltrials.gov. O.R.S.A. will continue to have discussions and provide data from the Canadian Rett syndrome Registry to help support the need for treatment options here in Canada.
Please join us at our Annual General Meeting tentatively scheduled for September 11, 2024 at 8pm for an open forum discussion.
We are stronger together! #ORSAstrong
O.R.S.A. Advocating for Canadian Families since 1991
Posted: 06/21/2024 by Steve
Many thank to the International Rett Syndrome Foundation @rettsyndrome.org for hosting the 2024 ASCEND family conference. It was wonderful and heartwarming to re-connect with families, friends, health care professionals and Industry. We are excited to announce our continued partnership with the IRSF, industry and new venders that can help our community.
Community Update
IRSF message “Change is required, courage is needed, community is the fuel, hope is mandatory” There are currently over 20 companies working hard on different drugs and therapies with the ultimate goal to cure Rett syndrome. Many new tools are available to support families on their website. Information provided from this conference will be available to families.
Acadia Pharmaceutical Update
The Canadian Pharma team is on track for Health Canada approval this fall. It’s exciting to know that the 1st drug approved for Rett could be available to Canadian’s as early as 2025.
O.R.S.A. is in the process of submitting an application to the Canadian Agency for Drug and Technologies in Health (CADTH) advocating on behalf of all the Rett associations in Canada and families to allow access to DayBue to Canadians.
Coming soon, education will be provided to medical professionals, families and caregivers. We suggested a tip sheet of ‘lived experience’ from families already on Daybue to reduce the likelihood of side effects. We will continue to ask for tools to help make this a smooth transition.
Taysha Gene Therapies Update
Our partnership with Taysha has been very successful and continues to be positive and fruitful as we paved the way for the 1st gene therapy trial to be available to adults in Montreal, Canada. Many thanks to Dr. Rossignol and team for trailblazing this potential treatment!
Anavex Life Sciences Update
Anavex appreciates all the Canadian families taking part in their clinical trials. As this trial comes to an end, they have informed us that other drugs trials are coming down the pipeline.
Neurogene Update
16 participants will be enrolled in total from the following sites, US, UK, and AUS. More information about this trial can be found on clinicaltrials.gov. O.R.S.A. will continue to have discussions and provide data from the Canadian Rett syndrome Registry to help support the need for treatment options here in Canada.
Please join us at our Annual General Meeting tentatively scheduled for September 11, 2024 at 8pm for an open forum discussion.
We are stronger together! #ORSAstrong
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