Research & Clinical News
Batten Disease Research Updates with Dr. Ineka Whiteman | March Illuminator Edition
Another month has passed, which means it's time for research updates. BDSRA Foundation's Head of Research & Medical Affairs Dr. Ineka Whiteman breaks down Batten disease clinical program updates, research news & opportunities, and resources [...]
Joint Statement to the Global Batten Disease Community
Dear Batten community, Over the past few years, Lexeo Therapeutics has been involved in developing an AAV-mediated gene therapy program for treating CLN2 Batten disease. However, additional funding and resources are required for further studies, [...]
CLN2 Batten Disease Community Survey
CLN2 Batten Disease Caregiver Study Understanding the patient journey, treatment approaches, and potential therapeutic opportunities for CLN2 disease BDSRA is leading a new survey-based study and invites all parents and caregivers (current or bereaved) [...]
Batten Disease Research Updates with Dr. Ineka Whiteman | February Illuminator Edition
Another month has passed, which means it's time for research updates. BDSRA Foundation's Head of Research & Medical Affairs Dr. Ineka Whiteman breaks down Batten disease clinical program updates, research news & opportunities, and resources [...]
Joint Statement to the Global Batten Disease Community
On Thursday, February 15, 2024, important news broke regarding CLN1 clinical research that global Batten disease patient advocacy groups wish to share with the Batten community. A patient with CLN1 disease was treated with the [...]
Batten Disease Q&A | 2024 WORLDSymposium Recap
Amy Fenton Parker It was a busy week for BDSRA President & CEO Amy Fenton Parker, traveling from Columbus, Ohio to Philadelphia for the quarterly BDSRA Board of Directors [...]
Joint Statement to the Global Batten Disease Community
Dear Batten community, As many of you know, we have been awaiting further news regarding the future of the CLN3 and CLN6 gene therapy clinical programs led by Amicus Therapeutics. Today, we wish to share [...]
Batten Disease Research Updates with Dr. Ineka Whiteman | January Illuminator Edition
Another month has passed, which means it's time for research updates. BDSRA Foundation's Head of Research & Medical Affairs Dr. Ineka Whiteman breaks down Batten disease clincial program updates, resources, and publocation summaries in her [...]
2023 Research in Review Webinar Recording | Batten Disease
Hosted by BDSRA's Head of Research & Medical Affairs, Dr. Ineka Whiteman, this webinar recaps Batten disease research from 2023. Dr. Whiteman is joined by a panel of fellow researchers and clinicians Follow BDSRA on [...]
Batten Disease Research Updates with Dr. Ineka Whiteman | November Illuminator Edition
NCL RESEARCHERS CHALLENGE 2023: Meet Dr. Joshua Dearborn and the WashU team As part of this year’s Annual Family Conference program in July, we hosted the inaugural NCL Researchers Challenge. Batten disease researchers from all [...]
REGENXBIO Webinar Recording | Batten Disease
BDSRA Head of Research & Medical Affairs Dr. Ineka Whiteman provides the latest news on REGENXBIO’s Batten disease programs, which is followed by a Q&A. She is joined by BDSRA board members Dr. David Pearce [...]
Joint Statement to our Worldwide Batten Community
As part of its third-quarter earnings release on Wednesday, November 8, 2023, REGENXBIO announced a corporate restructuring that included the difficult decision to halt the development of multiple potential AAV gene therapies, including the RGX-181 [...]
Batten Disease Research Updates with Dr. Ineka Whiteman | October Illuminator Edition
FEATURE ARTICLE: NCL 2023 Congress Research Report International Congress on Neuronal Ceroid Lipofuscinoses, Hamburg, Germany | September 26-30, 2023 Last month, BDSRA Head of Research & Medical Affairs Dr. Ineka Whiteman attended NCL 2023 with [...]
Batten Disease Q&A | 2023 NCL Congress Summary
The Batten Disease Support, Research, & Advocacy (BDSRA) Foundation was represented at this year's 18th International Congress on NCL in Hamburg, Germany by Head of Research & Medical Affairs Dr. Ineka Whiteman and President & [...]
Research Updates with Dr. Ineka Whiteman | September Illuminator Edition
Greetings from the NCL 2023 Congress in Hamburg, Germany! This month’s research column is hot off the press from the premier international research meeting for Batten disease. With over 200 in-person delegates including scientists, clinicians, [...]
Research Updates with Dr. Ineka Whiteman | August Illuminator Edition
NCL RESEARCHERS CHALLENGE – CONGRATULATIONS TO OUR WINNERS! As part of this year’s Annual Family Conference, we hosted the inaugural NCL Researchers Challenge. Batten disease researchers from all over the world were invited to enter [...]
Clinical Trial Updates | Gene Therapy for CLN2 Batten Disease
By Dr. Ineka Whiteman, BDSRA Foundation Head of Research & Medical Affairs BDSRA Foundation would like to share the latest developments in REGENXBIO's first-in-human gene therapy programs for the treatment of CLN2 Batten disease. Earlier [...]
Research Updates with Dr. Ineka Whiteman | July Illuminator Edition
FEATURED ARTICLE Annual Family Conference 2023 – Clinical & Scientific Program Highlights I’m delighted to share some of the highlights and key takeaways from this year’s Clinical & Scientific program. Dr. Jon Cooper, Dr. Dave [...]
