Rare Disease Advisor

A Patient With Pompe Disease Asks You to Take a Walk in His Shoes

If health care professionals could experience Pompe disease firsthand, they’d learn more than any textbook or lab result ...
BIOENGINEER

One Dose of a New Immunotherapy Combo Triggered Four Organ Crises in a Lung Cancer Patient

Immunotherapy has transformed the outlook for many people with advanced cancer, but a remarkable case report from clinicians in Shanghai shows just how sharply that power can cut both ways. A ...
BIOENGINEER

When Steroids Fail: Drug Combo Rescues Cancer Patient From Deadly Immunotherapy Heart Complication

Immune checkpoint inhibitors have transformed modern oncology, unleashing the immune system against tumors that once seemed untouchable. But the same biological firepower that destroys cancer cells ...
Labroots

Cold Burns Fat through Creatine, Not Just UCP1

A recent study showed that a fat protein called ALKHB5 drives cold-induced fat burning for heat production through ...
PR Newswire

BioMarin Announces Five-Year Phase 3 Data for POMBILITI® (cipaglucosidase alfa-atga) + OPFOLDA® (miglustat) at World Muscle Society Annual Congress

BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced new data evaluating the efficacy and safety of POMBILITI® ...

BioMarin Pharmaceutical Inc.: BioMarin Announces Five-Year Phase 3 Data for POMBILITI (cipaglucosidase alfa-atga) + OPFOLDA (miglustat) at World Muscle Society Annual Congress

Long-term data reinforce durable efficacy and safety of POMBILITI + OPFOLDA in late-onset Pompe disease SAN RAFAEL, Calif., Oct. 2, 2026 /PRNewswire/ -- BioMarin Pharmaceutical ...