RGX-181 and RGX-381 are potential one-time AAV Therapeutics for the treatment of the CNS and ocular manifestations of CLN2 disease, the most common form of Batten disease Patient dosed under a ...

LTS-101 is a one-time AAV gene therapy candidate intended to provide durable expression of the TPP1 enzyme in the central nervous system (CNS) of children with CLN2 diseaseFDA designations underscore ...

Most people haven’t heard of the CLN2 Batten Disease, an illness that ravages the motor skills, speech and vision of its host. Those who have the illness – mostly children – have a greatly shortened ...

A fundraiser for a rare neurodegenerative disorder affecting a four-year-old girl with a connection to racing was highlighted during the 2025 Indianapolis 500. Helen’s Pink Sky Foundation was featured ...

A new study shows that delivery of gene therapy to correct the gene mutations that cause CLN2 disease, or Batten disease, directly into the cerebrospinal fluid (CSF) has potential therapeutic effects.

A new study shows that delivery of gene therapy to correct the gene mutations that cause CLN2 disease, or Batten disease, directly into the cerebrospinal fluid (CSF) has potential therapeutic effects.

VICTORIA, Minnesota (WCCO) -- A Victoria, Minnesota, family is channeling their heartbreak into hope. When Dan and Stephanie Born welcomed their daughter Helen into the world, they felt their family ...

DelveInsight’s, “Batten Disease- Pipeline Insight, 2025” report provides comprehensive insights about 10+ companies and 12+ pipeline drugs in Batten Disease pipeline landscape. It covers the pipeline ...

SAN RAFAEL, Calif., July 24, 2024 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (BMRN) today announced that the U.S. Food and Drug Administration (FDA) has approved the company's supplemental Biologics ...

COLUMBUS, Ohio, Sept. 3, 2025 /PRNewswire/ -- Andelyn Biosciences, Inc., a pioneering and patient-focused cell and gene therapy Contract Development and Manufacturing Organization (CDMO), has ...

Despite a fatal, genetic children’s disease tearing through the fabric of the Bowman family, it was no match for their love, commitment and resilience. Bekah Bowman, a Boise, Idaho, mother and author ...