Brineura (cerliponase alfa) stabilized the progression of neuronal ceroid lipofuscinosis type 2 (CLN2) —…
Articles by Steve Bryson, PhD
Atypical neuronal ceroid lipofuscinosis type 2 (CLN2) — a type of Batten disease…
Amicus Therapeutics‘ investigational gene therapy AT-GTX-501 stabilized motor and language function in children with variant…
An examination of the spinal cords of mice that lack the PPT1 protein, the underlying cause of…
Treating a juvenile Batten disease mouse model with antisense oligonucleotides to correct its…
The proteins CLN6 and CLN8 — which are deficient in people with two forms of…
Common, non-invasive imaging techniques and a walking test used to assess patients with Batten disease…
A previously unidentified variant of a gene associated with adult-onset Batten disease (ANCL)…
Neurogene, which is developing therapies to treat people with Batten disease, has joined with…