Family calls for long-term access to 'miracle' drug
The father of a nine-year-old girl with a rare disease likened to childhood dementia has called for health bosses to prolong ...
Mothers rally and urge action over access to drug
Dozens of campaigners were in Westminster on Wednesday after being told NHS access to the drug was set to end in May. Health ...
Threat to son's treatment for rare disease 'agony'
A mother has spoken about her "painful" experience over the potential end to her son's life-enhancing treatment for a rare ...
Newcastle family fighting for Batten disease drug guarantee say surviving daughter is proof it works
Jessica Rich, nine, has a very rare and serious illness called Batten disease, but her life-saving drug could be about to be ...
What is Batten disease? Doctor and family share insights on rare condition
A local family is battling a rare disease diagnosis. The American pro wasn't a Swiftie when they met, but he sure is now. The Oscar-winning actor and his wife of over 30 years were reportedly found ...
The Reporter12y
Vacaville brothers Zack and Jakob Sharp to take…
The two boys will be participating in the 5K along with their cousins, Korry Sharp, 19, and Rachael, 16, who both have Finnish Late Infantile Batten Disease. For the boys, it”s a chance to spend ...
ITV3d
Batten Disease: Parents fight for costly drug for children's rare disease to remain on NHS
Siblings Ollie, 14, and Amelia Carroll, 12, both have Batten Disease, which is an incurable genetic disorder that causes rapid decline in a child's ability to walk, talk and see. Even though the ...
BBC23d
Parents' fears over loss of Batten disease drug
CLN2 Batten disease is an incurable genetic disorder that causes rapid decline in a child's ability to walk, talk and see. Phoebe, from Coventry, said daughter Flory's doses of Brineura ...
Neurogene: Potential To Target Root Cause Of Rett Syndrome With NGN-401
NGNE to update on a potential registrational phase 3 study, using NGN-401, for the treatment of patients with Rett Syndrome.
BBC3d
Threat to son's treatment for rare disease 'agony'
Her son, Isaac, eight, had CLN2 Batten disease diagnosed in August 2021. It is an incurable condition which causes childhood dementia, epilepsy and loss of sight. Ms Tilley said: "It is agonising.