TA-ERT is a fusion protein comprised of recombinant human NAGLU and a truncated human IGF-2 connected via an amino acid linker ... The incidence is about 100,000 and 200,000 live births. The disease ...

The company hopes to push the rare-disease treatment toward FDA approval next year. Four months after ditching its lead drug, ...

With Easter just around the corner, a host of Macon County groups are hosting Easter egg hunts and other special events.

EDEN PRAIRIE, MN / ACCESS Newswire / April 9, 2025 / Today, the Born and Schneider family announced the launch of Helen's Pink Sky Foundation, a new nonprofit dedicated to accelerating research, ...

Sangamo Therapeutics, Inc.'s $18M Eli Lilly deal boosts CNS genomic medicines. Fabry gene therapy data in 2025 is key for ...

Pressure on riskier credit is likely to continue, especially if companies with relatively high leverage react to the new ...

Kolkata: A three-year-old girl waiting for treatment for a rare disease died without getting any medical assistance under the ...

Trump tariffs live: US stocks plunge on fears of economic hit from tariffs on facebook (opens in a new window) Trump tariffs ...

“Diagnosing Gaucher disease typically involves a combination of clinical evaluation, family history, and laboratory tests. A blood test to measure glucocerebrosidase enzyme activity is crucial in ...

The official Foreign, Commonwealth and Development Office X account (@FCDOtravelGovUK) posted a warning today (April 2), ...

"Roads have been damaged and we need help with more machines so that we can clear the streets," Paros' mayor Costas Bizas told public broadcaster ERT. "All this catastrophe happened in two hours." ...

A live webcast of the fireside chat can be accessed from the Investor Relations section of Inozyme’s website under events, where a replay of the event will also be available for a limited time.