LOGO
LOGO

REGENXBIO's One-time Gene Therapy For Hunter Syndrome - History In The Making?

By RTTNews Staff Writer   ✉   | Published:   | Follow Us On Google News
rttnewslogo20mar2024 lt

Mucopolysaccharidosis type II (MPS II), also known as Hunter syndrome, is a devastating rare lysosomal storage disorder caused by a deficiency in the lysosomal enzyme IDS. It typically affects boys as young as 18 months. The most common symptoms include a rapid decline in learning and memory abilities, issues with heart and lung function, hyperactivity and behavioral difficulties, bone and joint deformities, and hearing loss.

The stock we are bringing to your attention today is that of REGENXBIO Inc. (RGNX), a clinical-stage biotechnology company, developing a gene therapy and one-time treatment for MPS II.

Subscribe to continue reading the article.
This article is part of our premium content offering.Subscribe with a RTTNews subscription.
Subscribe Now
Already subscribed? Sign in

For comments and feedback contact: editorial@rttnews.com

Global Economics Weekly Update: April 20 – April 24, 2026

April 24, 2026 15:15 ET
Economics news flow was relatively light this week even as the conflict in the Middle East continued, raising concerns for policymakers. In the U.S., spending data, initial jobless claims and pending home sales were the highlights. Business confidence in the biggest euro area economy was in focus in Europe. Inflation data from Japan gained attention in Asia.

Latest Updates on COVID-19