Research & Clinical News
BDSRA Applauds Latest News From ForeBatten Foundation on Zebronkysen
We applaud the news from our friends at ForeBatten Foundation that the FDA has provided IND clearance for the clinical investigation for Zebronkysen (FBF-001) under the N-of-1 guidance for individualized Antisense Oligonucleotide (ASO) drug products. [...]
Batten Disease Research Updates with Dr. Ineka Whiteman | August 2024 Illuminator Edition
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 in her monthly column, as [...]
Tern Therapeutics Enters into a Global Licensing Agreement with REGENXBIO Inc. for RGX-381 and RGX-181
Tern Therapeutics has announced its launch and entered into a global licensing agreement with REGENXBIO for RGX-381 and RGX-181 (now designated TTX-381 and 181, respectively) to form its initial therapeutic pipeline. TTX-381 and TTX-181 [...]
Global Update – CLN5 Batten Disease Clinical Trial
August 12, 2024 – On Friday, August 9, Neurogene Inc. announced enrollment is now complete for its Phase 1/2 investigational trial of NGN-101 Gene Therapy for the treatment of CLN5 Batten disease, with a total [...]
Batten Disease Research Updates with Dr. Ineka Whiteman | July 2024 Illuminator Edition
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 in her monthly column, as seen [...]
FDA Approves BioMarin’s BRINEURA® for Children Under 3 Years with CLN2 Batten Disease
Read the following press release and information below courtesy of BioMarin. Read the original press release by clicking here. SAN RAFAEL, Calif., July 24, 2024 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced that the U.S. [...]
BREAKING: BDSRA Foundation Unveils CoE & Affiliates; Batten Disease Global Research Initiative
ST. LOUIS (July 15, 2024) – The BDSRA Foundation announced its Clinical Center of Excellence & Affiliate members and the formation of the Batten Disease Global Research Initiative during Saturday night’s Dinner Dance at [...]
Batten Disease Research Updates with Dr. Ineka Whiteman | June Illuminator Edition
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 in her monthly column, as seen [...]
Batten Disease Research Updates with Dr. Ineka Whiteman | May Illuminator Edition
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 in her monthly column, as seen [...]
Batten Disease Research Updates with Dr. Ineka Whiteman | Post-April Illuminator Edition
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 in her monthly column, as seen [...]
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 [...]
