In patients with impaired renal function or fast-deteriorating eGFR at baseline, lucerastat was associated with a marked ...

Amicus Therapeutics FOLD has made solid progress with its lead marketed product, Galafold (migalastat), which generates the ...

Protalix ( ($PLX) ) has shared an update. On January 5, 2026, Protalix BioTherapeutics released a letter to stockholders outlining its strategy to ...

- U.S. Food and Drug Administration (FDA) provides a clear regulatory pathway to Accelerated Approval for isaralgagene civaparvovec using data from ongoing Phase 1/2 STAAR study, avoiding requirement ...

PHILADELPHIA--(BUSINESS WIRE)--Exegenesis Bio, a clinical stage global gene therapy company is pleased to announce that US FDA has granted Orphan Drug Designation (“ODD”) to EXG110, a novel gene ...

Brian Jones knows when it’s time for his treatment. Fatigue and pain remind him even if the calendar does not. But Brian, an Asheville physician specializing in nephrology, is grateful that his ...

Sustained benefit demonstrated with elevated expression of alpha-galactosidase A (α-Gal A) activity maintained for nearly four years for the longest treated patient as of the data cutoff date Positive ...

Shares of Sangamo Therapeutics (NASDAQ:SGMO) traded higher in the premarket on Tuesday after the company announced positive initial results from its pivotal Phase 1/2 STAAR study for its gene therapy ...

Fabrazyme (agalsidase beta) is a prescription drug that’s used to treat Fabry disease. The drug is given as an intravenous (IV) infusion by a healthcare professional. You’ll typically receive a dose ...

Fabrazyme (agalsidase beta) is a brand-name drug prescribed for Fabry disease in adults and some children. Fabrazyme comes as an IV infusion that’s given by a healthcare professional. The dosage ...