Enzyme replacement therapy effectively and safely reduces neuropathic pain in patients with Fabry disease, results of a double-blind placebo-controlled clinical trial show. The therapy also corrects ...

SUDO-550, an oral therapy for MS, was found to be safe and to reach the brain in its first human clinical trial, trial ...

Cornell researchers have developed an implant system that can treat type 1 diabetes by supplying extra oxygen to densely packed insulin-secreting cells, without the need for immunosuppression. The ...

This granted U.S. patent represents an important milestone for JayaBio,” said Pawel Krysiak, President and Chief Executive Officer of Jaya Biosciences. “It underscores our commitment to addressing the ...

Purpose: Enzyme replacement therapy (ERT) is a promising therapeutic intervention for lysosomal storage diseases. Posttranslationally engineered human β ...

ST. LOUIS--(BUSINESS WIRE)--M6P Therapeutics (“M6PT” or “the Company”), a privately held life sciences company developing next-generation recombinant enzyme and gene therapies for lysosomal storage ...

BioMarin Pharmaceutical BMRN reported third-quarter 2025 adjusted earnings per share (EPS) of 12 cents against the Zacks ...

Stem cells transplanted in utero (green) engrafted into fetal mouse brain tissue. Image credit: Q-H Nguyen/MacKenzie lab. Administering stem cell or enzyme therapy in utero may be a path to ...

It’s a big day for Sanofi as the pharmaceutical giant welcomes two European drug approvals, both for rare enzyme deficiencies. One regulatory nod ushers in the first treatment in Europe for a ...

As the name implies, a rare disease is an illness that affects far fewer people than more common conditions, like breast cancer or heart disease. Today, however, 7,000 rare conditions have been ...

After Intellia Therapeutics paused a pair of phase 3 trials for its CRISPR therapy in response to a liver safety signal, the ...

A team of researchers from the Hebrew University of Jerusalem, the Weizmann Institute of Science, and the University of Tokyo has made a significant breakthrough in the fight against cancer by ...