PPT1 is a recombinant protein used to treat an ultra-rare disease called Batten disease CLN1 for which there is ...
Doctors told Gordon Gray his daughters would not live to see their 12th birthday — -- The Hollywood couple who started a foundation to quickly fund research to find a cure for Batten Disease, ...
SA family of 4-year-old girl with terminal disease, needs community's support for gene therapy trial
SAN ANTONIO — Charli is fighting an extremely rare and terminal condition often referred to as childhood dementia at just 4-years-old. Charli qualifies for a gene therapy trial in California later ...
LYON, FRANCE and AUSTIN, TX / ACCESS Newswire / May 13, 2025 / Theranexus, a biopharmaceutical company developing drug candidates for rare neurological diseases, and the Beyond Batten Disease ...
EDEN PRAIRIE, MN / ACCESS Newswire / April 9, 2025 / Today, the Born and Schneider family announced the launch of Helen's Pink Sky Foundation, a new nonprofit dedicated to accelerating research, ...
Olivia Burtwistle is a 12-year-old girl in search of a cure for Batten disease. — -- Olivia Burtwistle grew as a typically curious and energetic child until soon after her fourth birthday, when ...
The National Institute of Neurological Disorders and Stroke has awarded a $3,953,139 SBIR grant to Collaborations Pharmaceuticals, Inc. for a Rare Disease RALEIGH ...
“CLN6 Batten disease is a devastating disorder with no approved therapies, and families deserve hope,” said Dr. Raymond Wang, Director of the Campbell Foundation of Caring Lysosomal Program at CHOC.
Nine year old Jeffrey Charlesworth from Accrington, who has Batten disease, spent his first respite weekend at Derian House Children's Hospice ...
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