A natural history study in people with CLN7 disease is now fully enrolled, but patients with CLN5 disease — each a…
Articles by Patricia Inácio, PhD
Ahead of this year’s Rare Disease Week on Capitol Hill, held virtually July 14–22, the EveryLife Foundation will…
More than half of the people with rare diseases and their caregivers, asked in a survey, were undecided or less…
The EveryLife Foundation for Rare Diseases is accepting applications for a scholarship program that aims to help adults with a…
The Scottish Medicines Consortium (SMC) has recommended that Brineura (cerliponase alfa) — the first and only…
A case report has described a rare case of a newborn diagnosed with CLN2 disease, or late infantile Batten disease, after…
The U.S. Food and Drug Administration (FDA) has designated Neurogene’s gene therapy, which aims to deliver a working copy of…
The number of treatments for children with rare diseases has grown over the past decade, according to a new study. However,…
Treatment with galactosylceramide (GalCer) — a type of fat molecule — improved motor function, reduced neuronal loss and increased lifespan…
Amicus Therapeutics‘ investigational gene therapy for CLN6 Batten disease may halt disease progression in children, according to interim results…