clinical trials

May 27, 2019May 24, 2019

Agency Unveils RaDaR to Help Patient Groups Develop Rare Disease Registries

News

RaDaR, the catchy new name for the U.S. government-run Rare Diseases Registry Program, aims to help patient advocacy groups with limited resources build their own disease registries. The site ... Read more

May 24, 2019May 24, 2019

FDA Clears ABO-202 Gene Therapy Candidate for Clinical Trials in Infantile Batten Disease

News

The U.S. Food and Drug Administration (FDA) accepted Abeona Therapeutics’ investigational new drug application for its one-time gene therapy candidate ABO-202 for children with infantile Batten disease, also known ... Read more

September 28, 2018September 28, 2018

Amicus Therapeutics’ Expanded Portfolio Includes Potential Gene Therapies for Batten Disease

News

Amicus Therapeutics has gained global development and commercial rights to 10 gene therapy programs for lysosomal storage disorders, including three potential first-to-market therapies for Batten disease. This pipeline expansion resulted from ... Read more

June 2, 2017

Brineura Approved as Batten Treatment in Both Europe and US as Clinical Studies Continue

News

Although Brineura (cerliponase alfa) is now approved both in Europe and the U.S. to treat CLN2 type Batten disease, its clinical trial path is far from over. As the approval rested on ... Read more

May 1, 2017May 1, 2017

FDA Approves First Treatment for Batten Disease Type CLN2

News

Patients with a specific type of Batten disease now have an approved treatment, with the U.S. Food and Drug Administration’s (FDA) recent approval of Brineura (cerliponase alfa) for use ... Read more

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