A father is appealing for donors as his teenage son faces a life-threatening blood disorder and needs a stem cell transplant within weeks to improve his chances of survival.

US FDA grants Breakthrough Therapy designation to Sanofi’s venglustat for type 3 Gaucher disease: Paris Sanofi announced that the US Fo ...

Gaucher disease is a rare inherited disorder in which fatty substances accumulate inside certain cells, gradually damaging organs such as the spleen, liver and bones. The condition occurs due to a ...

Sanofi has received Breakthrough Therapy designation from the US FDA for venglustat, an investigational oral drug targeting neurological symptoms of type 3 Gaucher disease, a rare genetic disorder ...

Gaucher is a rare genetic condition that causes fatty substance build up in the tissues of spleen, liver and bones and ultimately causes damage and dy.

Sanofi's venglustat earns Breakthrough Therapy designation in the US for type 3 Gaucher disease Paris, March 18, 2026. The US Food and Drug Administration (FDA) has granted Breakthrough Therapy design ...

The U.S. Food and Drug Administration has granted breakthrough therapy designation to Sanofi’s (SNY) venglustat, a novel, investigational oral glucosylceramide synthase inhibitor (GCSi), for the ...

Cardiovascular and cardiometabolic diseases are leading causes of morbidity and mortality worldwide, driven in part by chronic inflammation. Emerging research suggests that the bone marrow ...

Explore the critical issue of the Right to Life and access to treatment for rare diseases in India. Understand the struggles faced by families and the systemic challenges that delay crucial medical ...

Vice Adm. Robert Gaucher has begun his tenure as the Department of War’s first direct reporting portfolio manager, or DPRM, for submarines, a newly created leadership role to oversee the Navy’s ...