September Newsletter

BDSRA Board Corner

Press release from the Board of Directors of the BDSRA. Please read below.

We, the board of directors of the BDSRA, are collectively sending a message out to the community today to give you all an update on a few matters.

First, as many of you know, one of our founders and our Executive Director for over 25 years, Lance Johnston passed away recently. There are not enough words on this page to describe his impact on this organization. He was instrumental in the success and growth of BDSRA and in helping families with support and care for their children. To honor Lance Johnston and his family, we will be starting the Johnston Family Memorial Fund to be used to help support our Batten families in some capacity. We will give out more specifics on this at a later date as we develop this fund.

Second, the BDSRA would like to thank Tauna Batiste for her service to BDSRA and the Batten community. She has chosen to move on to pursue other opportunities, and BDSRA wishes her success in her future endeavors.

We’d like you all to welcome Michelle Churchill as our new interim Executive Director. Michelle will be at this position while we conduct a search for a permanent replacement. Many of you may know Michelle in her previous work with BioMarin, where she recently retired. She’s also been a volunteer at some of our previous conferences.

We would also like to introduce Morgan DeBoth. Morgan will be taking over the position of Director of Family Services. Morgan comes from a social work background and is committed to promoting equity and advocacy.

Other staff notes: Noreen Murphy has been promoted to Senior Director of Education and Advocacy. We thank her for her dedication to our families and her 6 years of service to the BDSRA. The BDSRA board also recognizes another staff member, Colin Montieth for his work as Development Coordinator.

Please welcome our new staff members Michelle and Morgan!

It is a perilous time in this country due to the COVID-19 pandemic and we also recognize the daily battle that is Batten Disease. We haven’t wavered from that mission to find a cure and we want you to know that we are united as ever in the fight against Batten Disease. The board of directors and the staff appreciates your efforts whether it’s hosting a virtual 5k or having your own fundraisers or volunteering. We know that your fight is our fight and together we can make this organization the best it can be.

If you have any questions for us regarding this news release please direct them to Trent Lewis by direct message or email. Trent is head of our communication sub committee for the board.

Thank you,
The Board of Directors BDSRA

Meet the Team: Morgan DeBoth Manager of Family Support

Morgan comes from a social work background and is committed to promoting equity and advocacy. She joins BDSRA with seven years of professional experience in the nonprofit field. She has advocated for individuals of all ages and brings with her a passion for serving the community.

Morgan received her undergraduate degree from Ball State University located in Muncie, IN. She began her work as a victim’s advocate for women and children whom experienced domestic violence and/or sexual assault. In 2016 she completed her certificated in Cognitive Behavioral Therapy from the Beck Institute. This inspired her to continue to advocate for healthy families and for better quality of life for all individuals. Her most recent position at Gladden Community House as the community liaison launched her into the Columbus community and allowed her to fundraise, network, write grants and advocate for the benefit of the local community and its members. Morgan currently sits on the board of the Franklinton Arts District and pours her heart into the advocacy of the arts as a healing medium to those whom are most at-risk.

Morgan brings her passion for serving others, fundraising, community connecting, and advocacy to her new role of Manager of Family Support with BDSRA. In her free time, she volunteers with her local community, takes watercolor paint classes, explores new hiking trails, and spends time in her vegetable garden

Meet the Team: Michelle Churchill Interim Executive Director

Michelle is honored to join BDSRA. You may recognize her and her family as volunteers at past conferences and participants in local races and fundraisers.

Michelle retired last year after a 28 year career in medical and biotech sales and corporate training. Through an earlier position with a pediatric company, she was asked to serve on the planning committee for the Central Ohio Chapter of the American Lung Association which was the beginning of her interest in fundraising.

Michelle spent 18 years with BioMarin where she developed a passion for rare disease, advocacy, and education with an emphasis on the family journey. Her experience in working with families lead her and her family to become active with BDSRA, The National MPS Society and other small local organizations. After retiring, she started substitute teaching outside of Pittsburgh and most recently was asked to develop and teach a professionalism curriculum for a technical school in Columbus.

Michelle received her undergraduate degree from The Ohio State University and is a lifelong fan. She and her husband Don have been married for 30 years and have two adult children, Lauren and Owen who also work in healthcare fields. Michelle and Don enjoy traveling and are working on a goal to visit all the presidential libraries, Big 10 stadiums, and major national parks.

She is looking forward to supporting the organization, staff, and families.

Upcoming Webinars & Conferences

Rare New England Annual Conference 2020

“Improving Health Care Experiences in the Rare Disease Community” is coming up October 24th. The link below has more information on the topics covered and how to register.
https://www.rarenewengland.org/rne-conference-2020.html

The Patient Experience: What is it Like Participating in a Clinical Trial?
Tuesday, Oct 20, 10:00 AM EDT

"After exploring the landscape of drug development in our first webinar, our second webinar will focus on what it means to participate in a clinical trial, with a focus on the patient's perspective.

It is crucial for patients and their families to understand what is involved, in addition to being aware of their rights and obligations, if they so choose to participate in a clinical trial."

https://www.bigmarker.com/bioalliance-video-network/The-Patient-Experience-What-is-it-Like-Participating-in-a-Clinical-Trial

Influencing State Policy: Rare Disease Advisory Councils Pave the Way!

Oct 13, 2020 02:00 PM EDT

"NORD's Policy Team is excited to provide an update on one of NORD’s main policy priorities at the state level: Rare Disease Advisory Councils (RDACs). RDACs act as an advisory body, enabling a stronger voice for the rare disease community in state government. This webinar will provide an update on the existing 14 RDACs, detail NORD’s future work in this area, and discuss opportunities for our members to get more involved. In addition, NORD policy staff will provide an update on current policy happenings."

https://rarediseases.zoom.us/webinar/register/4215983900309/WN_PTRB-H-RRC2savomzgyt8Q

An Update from Amicus Therapeutics

The following is from their Press Release, to read more visit their website

Amicus Therapeutics Receives European Medicines Agency PRIME Designation for CLN6 Batten Disease Gene Therapy

CRANBURY, N.J., Sept. 24, 2020 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq: FOLD), a global, patient-dedicated biotechnology company focused on discovering, developing and delivering novel medicines for rare diseases, today announced the European Medicines Agency (EMA) has granted Priority Medicines (PRIME) designation to AT-GTX-501, the Company’s investigational gene therapy for children living with variant late infantile neuronal ceroid lipofuscinosis 6 (vLINCL6) disease, also known as CLN6 Batten disease.

The PRIME initiative provides enhanced support and increased interaction to developers of promising medicines with the goal of optimizing development plans and speeding regulatory evaluations. The goal of EMA’s PRIME is to help patients benefit as early as possible from innovative new therapies that have demonstrated the potential to significantly address an unmet medical need.

The PRIME designation is based on data from the ongoing Phase 1/2 clinical trial evaluating a single dose of AT-GTX-501 for the treatment of children with CLN6 Batten disease. Additional information about the trial is available at ClinicalTrials.gov (NCT02725580).

“We are very pleased that the EMA has recognized the potential of our CLN6 gene therapy. Based on our preliminary clinical data, we believe AT-GTX-501 could potentially be a transformative treatment option for children living with CLN6 Batten disease, an ultra-rare, debilitating condition that presents in early childhood and is often associated with childhood death,” said John F. Crowley, Chairman and Chief Executive Officer. “We look forward to continuing to work closely with the EMA to accelerate development of this first potential treatment option for children living with CLN6 Batten disease.”

Additional data from the ongoing Phase 1/2 clinical study will be presented at the Child Neurology Society Annual Meeting in October. Regulatory interactions are ongoing and the Company expects to provide feedback on the path forward in early 2021.

In the U.S., AT-GTX-501 previously was granted Rare Pediatric Disease and Orphan Drug designations by the United States Food and Drug Administration. In the EU, the Company now holds PRIME and orphan medicinal product designations.

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.

Faith King

January 23, 2014- September 21, 2020

Lydia Wahlstrom

July 18, 2004- August 12, 2020

Elijah Keller

March 19, 2006 - September 23, 2020