November 2020 Volume 31, Issue 11
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Board Corner: Introducing New Board Members for 2021
Meet our incoming BDSRA Board Members!
Rene Martinez
Rene is mom to Sofia, CLN3, and is from Washington State.
Please describe why you would like to be a member of the board.
"I appreciate the help I have received through BDSRA and see this as an opportunity to serve & give back. In the past I have been a board member of the Washington State Developmental Disabilities Council and our local ARC which is a service provider for those with disabilities. As a parent of an older batten child I feel I can bring experience and knowledge to the board."
What activities and strategies would you personally participate in to advance the organization (ex. Industry connections, fundraising, etc)?
"Sofia wanted to participate in pageants when she was 12. We couldn’t find any locally so she wanted to start one. I created a 501c3 for her Shining Star Pageant. It started with 14 girls and now has over 50 each year from all over the state. I am a detail oriented and schedule driven person. If I had a magic wand I would like to see BDSRA increase its personal relationships with families. I do see the need for research and industry connections but personally as a care provider it can be a long and lonely road. Recently we have seen all the way technology can be used. Since we have families all over the world we could use technology to facilitate support groups based on interest (type, moms, dads, behavior help, ask a doc etc). I also would like to educate families more on advocacy so they can speak locally to law makers and work to create better assistance programs in their own state. Advocacy skills could also be uses to help families navigate the challenges within the educational system."
Fred Surrey
Fred is a bereaved dad to Lauren and Michelle, CLN3, from Staten Island New York.
Please describe why you would like to be a member of the board.
"Since I have been part of the BDSRA family since 1999, I would like to be on the Board to give back to the organization all the love and support to newer families that was given to my family 20 years ago. The BDSRA gave my family a place to learn about the disease, to be with others who have gone through or were going through the same life experience as my wife and I were and to share the ups and downs of those experiences. I want to help make sure that vital help is there for any family with any version of NCL. I feel the need to help ensure that the organization continues its mission to support families and to fund research to help slow the progression of the disease and ultimately find a cure. Since Batten disease is considered one of the many "often diseases" it is vital that our organization continues to to advocate , grow and flourish to keep research moving forward, despite the lack of governmental funding help. In addition to helping the Board in whatever endeavors they entrust me with with i know I can help support newly diagnosed families by imparting to them many of the positive experiences as well as helping them avoid the negative experiences of my 20 plus years dealing with this disease."
What activities and strategies would you personally participate in to advance the organization (ex. Industry connections, fundraising, etc)?
"I believe I could help with methods of raising funds, tracking the income and expenses of the organization and I can deliver prepared speeches in front of large groups if the need arises."
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Research Updates
This month Neurogene announced the launch of a natural history study for CLN5 and CLN7, two subtypes of Batten disease. A natural history study tracks the course of a disease over time to understand how the disease progresses and may give insight into how the disease might be managed. Learn more: https://bit.ly/36E2e0o
Check out this new publication from Margaux C. Masten , Jonathan W. Mink & Erika F. Augustine from the University of Rochester Batten Center, Batten disease: an expert update on agents in preclinical and clinical trials
REGENXBIO third quarter operational highlights included...
"RGX-381 for the Treatment of Ocular Manifestations of Late-infantile Neuronal Ceroid Lipofuscinosis Type 2 (CLN2) Disease
- REGENXBIO is on track to submit an Investigational New Drug (IND) application for a Phase I/II study of RGX-381 in patients with CLN2 disease by the end of 2020 and plans to initiate enrollment in the first half of 2021.
- Data from non-human primates demonstrate elevated and sustained levels of TPP1, the enzyme deficient in patients with CLN2 disease, in the vitreous following a single subretinal injection of RGX-381.
RGX-181 for the Treatment of CLN2 Disease
- REGENXBIO expects to submit an IND for the intracisternal delivery of RGX-181 in the first quarter of 2021, and plans to initiate enrollment in a Phase I/II trial in the first half of 2021."
Learn more about updates in Amicus Therapeutics' CLN3, CLN6, and CLN1 programs from their third quarter financial results here!
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Introducing the Fore The Journey Fund!
The journey through Batten disease can be challenging and families cherish moments that bring happiness to their children. A visit to a zoo, sports lessons, or a swim in a community pool can provide much-needed smiles.
The Fore The Journey Fund is a joint collaboration with the BDSRA and the ForeBatten Foundation. This unique grant offers Batten families experiences, memberships, and gifts in the hopes of providing happy moments. Your request can be as individual as you! We are happy to consider any request that will bring smiles to your family.
For your inspiration we have listed some ideas:
- Season Passes to the Zoo, Aquarium, etc.
- YMCA, sports or gym membership
- Family Portrait session
- Adaptive Technology for your child
- Spa Day for Mom/Dad
- Airline Tickets for a friend or relative visit
Please visit our website to download the application 😄
https://www.bdsra.org/fore-the-journey-fund/
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This month we have new Facebook fundraisers organized by:
- Ashley Bieker
- Hannah Booth
- Rachel Cannon
- Krista Marie Cartwright
- Timothy Coon Jr.
- Casey Dewey
- Marcie Minter
- Emma Naji
- Jessica Storm
- Helen Vanderlan
- Drew Wiley
We are so excited each day to log in and see the list of your names growing. Thank you for sharing and supporting our mission on social media! You can visit each fundraiser by clicking on the names above.
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In Loving Memory
Remember with us those we have lost to Batten disease. It's in their honor and memory that we work every day to build a brighter future for families.
Mya Ogle
10/02/08-11/22/20
Adia Elfert
07/8/00-11/19/20
Willie Neifert
8/27/96-11/17/20
Lilli Summers
2/22/17-10/31/20
To have your loved one's name placed in the Illuminator please reach out to Morgan at mdeboth@bdsra.org.
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Your gift makes all the difference in the lives of families.
You can view recent donations to BDSRA here.
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Batten Disease Support and Research Association
(614) 973-6013 | info@bdsra.org | www.bdsra.org
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