Batten Disease News

January 13, 2022January 13, 2022

CLN6 Mutations May Lead to Batten Without Vision Loss, 3 Cases Show

Batten Disease News

Mutations in the CLN6 gene — which have been associated with different forms of Batten disease — may lead to the development of an atypical form of juvenile Batten that ... Read more

January 6, 2022January 6, 2022

Taysha Approved in Canada for Trial Testing TSHA-118 for CLN1 Disease

Batten Disease News

With approval from Health Canada, Taysha Gene Therapies will begin a Phase 1/2 clinical trial testing TSHA-118, its investigational gene therapy for CLN1 disease, also known as infantile Batten disease. This ... Read more

December 30, 2021December 30, 2021

Rarebase Launches Collaborative Platform to Find New Therapies

Batten Disease News

Rarebase, a public benefit biotechnology company, has launched the Function therapeutic discovery platform specifically for rare neurological diseases, including Batten disease. Led by scientists, engineers, patients, and advocates, Function combines ... Read more

December 23, 2021December 23, 2021

International Experts Develop Guidelines for CLN1 Management

Batten Disease News

An international team of experts has developed a set of consensus-based, family-informed recommendations for the diagnosis and best care management of neuronal ceroid lipofuscinosis type 1 (CLN1) disease. The guidelines ... Read more

December 16, 2021December 16, 2021

Foundation Helps Sponsor Drafting of Guidelines for Juvenile Batten

Batten Disease News

The Beyond Batten Disease Foundation (BBDF) is sponsoring Care Beyond Diagnosis in its efforts to draft international guidelines to help clinicians counsel and treat patients with CLN3 disease, or juvenile Batten ... Read more

December 9, 2021December 9, 2021

2 Scientists in Australia Awarded 2021 Batten Research Grants

Batten Disease News

Winners of the Australia and New Zealand Batten Disease Research Grant for 2021 have been announced by the Batten Disease Support and Research Association (BDSRA) Australia, the program’s sponsor. They are Anthony ... Read more

November 18, 2021November 18, 2021

CLN5 Gene Therapy Trial to Start Early Next Year in Rochester

Batten Disease News

Enrollment in a clinical trial testing NGN-101 — an experimental gene therapy for CLN5, a type of late-infantile Batten disease — is expected to start early next year at the ... Read more

November 11, 2021November 11, 2021

Rare Disease Groups in US Join in Plea for Care Across State Lines

Batten Disease News, COVID-19 News

Over 230 national organizations signed a letter urging all 50 U.S. state governors to “maintain and expand” flexibility with licensure requirements for the duration of the COVID-19 pandemic to ease ... Read more

November 4, 2021November 4, 2021

Gene Therapy for CLN2, LX1004, Supported by $100M in Financing

Batten Disease News

Lexeo Therapeutics announced that it raised $100 million in a financing round to help support the development of pipeline therapies, including LX1004, a potential gene therapy for CLN2 disease, also known as ... Read more

October 29, 2021October 29, 2021

NIH Study: Cost of Treating Rare Diseases ‘Large’ Relative to Other Ills

Batten Disease News

A retrospective study by the National Institutes of Health (NIH) suggests that healthcare costs for those with rare diseases have been underestimated, possibly being three to five times higher than ... Read more

October 28, 2021October 28, 2021

RARE-X, Global Genes to Help Collect Rare Disease Patient Data

Batten Disease News

In their continued efforts to improve health equity for people with rare diseases, Global Genes and RARE-X have joined forces to help advocacy groups collect patient data and make the ... Read more

October 21, 2021October 21, 2021

Global Genes, Diversity Coalition Team Up to Advance Health Equity

Batten Disease News

Global Genes has partnered with the Rare Disease Diversity Coalition (RDDC) to advance health equity for rare disease patients and caregivers in underrepresented communities of color. “For rare disease patients, ... Read more

October 14, 2021October 14, 2021

Taysha Secures Licence for UT Southwestern’s CLN7 Gene Therapy

Batten Disease News

Taysha Gene Therapies has secured an exclusive option from the University of Texas Southwestern (UTSW) to license worldwide rights to a clinical-stage gene therapy program for CLN7 disease, a form ... Read more

October 14, 2021October 14, 2021

Rare Disease Diversity Coalition Awards $600K to Combat Disparities

Batten Disease News, COVID-19 News

The Rare Disease Diversity Coalition (RDDC) awarded $600,000 in grants to ease the disparities faced by rare disease patients of color. These Impact Rare Disease Solution grants will go five RDDC ... Read more

October 7, 2021October 7, 2021

‘Rare’ Documentary in Kickstarter Campaign to Raise $45K by Oct. 28

Batten Disease News

A crowdfunding campaign aims to raise $45,000 to support “Rare,” a documentary film featuring the struggles and achievements of people living with rare diseases and their families. Sweis Entertainment and ... Read more