Treatment for approximately one year with Batten-1 (miglustat) — an experimental oral small molecule developed by Theranexus — in…
Articles by Margarida Maia, PhD
People with juvenile Batten disease treated with the small molecule Batten-1 (miglustat) for 18 months in a clinical trial,…
With its RareCare Patient Assistance Program, the National Organization for Rare Disorders (NORD) helps to cover healthcare costs that…
Regenxbio will not move forward with the development of RGX-181 and RGX-381, gene therapy candidates for the treatment of…
This year’s Orphan Drugs and Rare Diseases Conference — the 4th annual such event, taking place Oct. 9-10 in…
The University of Rochester Medical Center (URMC) has been awarded a funding boost and will expand its involvement in…
The Batten Disease Support and Research Association (BDSRA) is challenging people to walk, run, or bike a virtual…
A new mutation in the MFSD8 gene was identified as the cause of CLN7 disease, a form of late-infantile Batten disease, in…
With approval from Health Canada, Taysha Gene Therapies will begin a Phase 1/2 clinical trial testing TSHA-118, its investigational gene…
Winners of the Australia and New Zealand Batten Disease Research Grant for 2021 have been announced by the Batten Disease Support…