Research & Clinical News
Batten Disease Research Updates with Dr. Ineka Whiteman | February & March 2025
It's time for Batten disease 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 [...]
BREAKING: Batten Disease Global Research Initiative Announces Grant Recipients
We are proud to announce the recipients of the Batten Disease Global Research Initiative inaugural Research Grant round. Learn more about the recipients and their research by clicking the button below. Read the Full [...]
CLINICAL UPDATE: Tern Therapeutics Gives CLN2 Batten Disease Update
The following letter from Tern Therapeutics shares the latest on TTX-381 and TTX-181, gene therapies being developed for the treatment of CLN2 Batten disease. Read the PDF of this letter here. February 27, 2025 Dear [...]
Batten Disease Research Updates with Dr. Ineka Whiteman | January 2025
It's time for the first Batten disease research updates of 2025! BDSRA Foundation's Head of Research & Medical Affairs Dr. Ineka Whiteman breaks down Batten disease clinical program updates, research news & opportunities, and [...]
Batten Disease Research Updates with Dr. Ineka Whiteman | November 2024
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 [...]
Batten Disease Research Updates with Dr. Ineka Whiteman | October 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 [...]
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, [...]
