August 2021 Volume 32, Issue 8
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BDSRA Staff Attends World Orphan Drug Congress 2021
"The Word’s Most Important Orphan Drug & Rare Disease Event"
The 11th annual World Orphan Drug Congress USA is back — and in-person — with their most comprehensive program and inclusive gathering of rare disease stakeholders yet! Becoming the largest rare disease and orphan drug conference globally has allowed them to bring together leading industry, government and regulatory authorities, patient advocacy groups, payers, investors and solution providers. The World Orphan Drug Congress USA allows attendees to meet, brainstorm and dialogue on ways to advance orphan drug development and improve access to life-saving therapies.
Over 1,500 attendees from 50+ countries gathered in Oxon Hill, Maryland on August 25th-27th for the 11th edition of World Orphan Drug Congress USA! WODC has become the industry defining conference on Advanced Therapies for rare diseases, featuring discussions on clinical development, manufacturing and commercialization of cell and gene therapies. It also is the annual gathering for artificial intelligence, digital health solutions, and drug development platforms transforming the rare disease industry!
BDSRA CEO Amy Fenton Parker and Vice President of Support and Advocacy Morgan DeBoth attended the 3-day conference to learn about clinical updates, treatments, advocacy efforts and information in the Orphan Disease space.
To learn more about #WODCUSA, check out the highlight on our instagram or click the button to visit the official website:
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BDSRA is looking to add parent and non-parent board seats:
Applications are due August 31th
To see a full description of roles and responsibilities please click here.
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Do You Follow BDSRA on Social Media?
Want to see more BDSRA content? Don't miss out on upcoming events, information, campaigns and community posts! Follow @BDSRA on Facebook, Instagram, LinkedIn and Twitter to stay in-the-know on all things happening in our Batten Community!
Click below to follow all of our accounts:
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Upcoming Events for Batten Families
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Join us for the Ask-An-Expert: Back To School Edition where you can ask a teacher questions about your child's IEP, 504 Plan, and how to support siblings as they transition to the next school year.
To register for this event or to send this event to other families, please click here:
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New dates for NCL 2021! The International Congress on Neuronal Ceroid Lipofuscinosis event will take place at Washington University, St Louis, October 6-10, 2021 and will be a 'hybrid' conference. This means both in-person and online-only registration options will be available. There is also a reduced registration fee for families available!
This a parent-friendly conference; decoding the science for everyone to understand. BDSRA's Amy Fenton Parker and Morgan DeBoth will be in attendance. All are welcome!
More details can be found by visiting: www.ncl2021.org
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RARE Patient Advocacy Summit 2021: Virtual | September 27-29, 2021
Each year, Global Genes convenes one of the world’s largest gatherings of rare disease patients, caregivers, advocates, healthcare professionals, researchers, partners and allies. Join us for a variety of interactive and educational events, meet-ups, workshops and networking opportunities. Here you’ll have the opportunity to connect and engage with others in the rare disease community, while experiencing the sense of community and belonging we’ve missed so much. Gain insights about the latest in rare disease innovations, best practices for advocating on an individual and organizational level, and actionable strategies you can implement immediately to accelerate change. Registration is now open!
To learn more information or to register, click the button:
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NORD Rare Summit 2021: Virtual | October 18-19, 2021
Each year, the NORD Summit, one of the largest multi-stakeholder events in rare disease, brings together participants spanning the rare community, including rare disease experts and leaders from patient advocacy groups, government, industry, and academia to discuss the most current and critical topics related to rare diseases and orphan products. Registration is now open!
To learn more information or to register, click the button:
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Join this Batten Family's Fundraiser "Virtually" or In-Person!
"These rides are inspired by my two courageous and amazing nephews. They are Titus and Ely and they both have Batten disease. Titus ran into the arms of Jesus at the age of 6. His parents, Danny and Bekah and little brother, Ely, miss him tremendously. Just 2 weeks after Titus passed, Ely had the life giving opportunity to begin an enzyme replacement treatment. This incredible treatment was created by a team of experts who desire to help Batten children have a prolonged and high quality of life. Ely is living proof that the research being done is indeed working! He is still able to walk, eat, and communicate.
This would not be possible without this enzyme replacement therapy. We are forever grateful for the work being done. The research teams and doctors are currently in the midst of finding a treatment to prevent blindness in children as well as gene therapy. There is a lot more work to be done and it is exciting! All funds raised during Bike for Batten will go directly to BDSRA (Batten Disease Support and Research Association). You WILL make a difference in the lives of these amazing children."
-Bekah Murphy, Bike For Batten
Join us in raising money for Batten Disease research by Biking for Batten!
Register below for either of our two Bike for Batten events.
Full Rides/Partial rides/No biking options available.
Ride 1: Friday, September 17th- 9am @ Yep Kanum Park
in Colville, WA (18mi)
Ride 2: Sunday, September 19th- 2pm @ Northwest Nazarene University in Nampa, ID (20mi)
You can also choose to ride from your hometown or just donate!
You can also purchase a Bike For Batten T-Shirt and support the cause by clicking here.
Register or donate by clicking the button:
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A Note from Industry Partners
During the 2nd Quarter 2021 Financial Results and Operational Highlights call on August 9th 2021, RGNX shared the following:
Gene Therapy Using NAV Vectors for Rare Genetic Diseases
RGX-181 for the Treatment of Late-infantile Neuronal Ceroid Lipofuscinosis Type 2 (CLN2) Disease
REGENXBIO plans to provide a program update by the end of 2021.
RGX-381 for the Treatment of Ocular Manifestations of CLN2 Disease
REGENXBIO plans to provide a program update by the end of 2021.
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Natural History Study enrolling in person.
If you have concerns about traveling, please reach out to Morgan DeBoth at mdeboth@bdsra.org to coordinate alternative participation opportunities.
https://clinicaltrials.gov/ct2/show/NCT03822650?cond=cln5&draw=2&rank=1
Caregiver Focus Groups for CLN5
Dates will be determined by the families’ availability. View article here.
Neurogene announces the European Medicines Agency (EMA) has granted Orphan Drug Designation (ODD) to CLN5 Batten Disease Therapy. This announcement follows the FDA’s Orphan Drug Designation which demonstrates continued progress in establishing a Batten disease franchise.
To read the full press release click here.
Neurogene is currently working with RTI Health Solutions (RTI‑HS), an independent, nonprofit research organization to better understand the disease progression of Batten disease subtype CLN5 from the caregiver’s perspective.
Caregivers of children (either currently living or deceased within the past 12 months) with Batten disease subtype CLN5 who are eligible for this study (Qualitative Interviews With Caregivers of Children With CLN5-Specific Batten Disease) and live in the United States will be invited to participate in a telephone interview with two RTI‑HS interviewers lasting approximately 90 minutes. Interview participants will receive an electronic gift card (e.g., Visa, Amazon, or other online store of choice) for $150 in appreciation for their time.
During the interviews with RTI‑HS, caregivers will be asked to describe their child’s and their own experiences surrounding the journey to and following diagnosis, including observed signs and symptoms of the disease, delays in any of the developmental milestones, and perceived impacts of Batten disease subtype CLN5 on the child, caregiver, and family.
The discussion will be audio recorded and transcribed to ensure the interviewers capture the important points that participants share and to help write a report summarizing the results of the interviews. Participants’ names and any other identifying information will not be associated with the transcripts. The audio recordings will be destroyed once a final report is written.
If you are interested in participating in a telephone interview, please call Danielle at L&E Research at 720-647-3228 who will ask you a few questions to confirm that you qualify for this study. If you qualify, L&E Research will schedule you for an interview at a day and time that is convenient for you.
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Research Study Opportunity
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August 2021 Facebook Fundraiser Spotlight:
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. Thank you!
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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.
To have your loved one's name placed in the Illuminator please reach out to Morgan at mdeboth@bdsra.org.
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Thank You BDSRA Community of Donors!
Thank you to the donors last month who gave so generously to honor and in memory of loved ones and our community as a whole. We want to acknowledge you for your contributions. They allow us to keep serving families and advancing the science of a meaningful treatment. Your dollars and support have helped create the leading patient organization in the Batten disease community, and we are dedicated to continue that legacy of children and families
You can view recent donations to BDSRA
by clicking here.
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Your gift makes all the difference in the lives of families.
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Batten Disease Support and Research Association
(614) 973-6013 | info@bdsra.org | www.bdsra.org
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