Author Archives: Forest Ray PhD

Twins’ Different Symptoms Complicate Diagnosis

Juvenile Batten disease is difficult to diagnose promptly and accurately, as demonstrated by the case of twin girls with different symptoms of the same disorder. The case suggests that a referral to a specialist should be made when a patient’s clinical picture doesn’t line up with their history, or…

EMA Names Potential Gene Therapy for CLN5 Batten an Orphan Drug

The European Medicines Agency (EMA) designated Neurogene’s gene therapy candidate for CLN5, a form of late-infantile Batten disease, an orphan drug as a potential treatment of the disorder. “This regulatory designation is an important milestone toward addressing the urgent, unmet need of families and patients living with CLN5, and…

Updates Delayed on Regenxbio’s CLN2 Gene Therapy Programs

Regenxbio plans to provide updates on its RGX-181 and RGX-381 gene therapy candidates for late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease, one of the most common forms of Batten disease, in the second half of this year. The company is currently reviewing information requests from…