Amicus Therapeutics’ first oral drug for the rare condition Fabry disease has been approved by the FDA, giving patients an alternative to conventional injected enzyme replacement therapy.

This came after Takeda Biopharmaceuticals presented the proposal for an update in the package insert regarding home infusion ...

Market research firm Optima Insights has predicted that sales of Fabry disease drugs will more than double from around $1.8 billion last year to $3.8 billion by 2027, driven by new oral drugs such ...

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.

Sangamo Therapeutics shares rose after the company said the outcome of a recent successful interaction with the Food and Drug Administration ... or ST-920, for Fabry disease.

Mr Mikhaiel claims that if he were sent back to Egypt, he would not be able to access a drug called migalastat, which is used in Scotland to treat Fabry disease. In a letter seen by BBC Scotland ...

Fabry's disease is an X-linked disorder caused by ... Genzyme's Fabrazyme and Transkaryotic Therapies' (TKT) Replagal. Both drugs are formulations of α-galactosidase, but are produced in ...

Fabry disease in an inherited condition in which ... It confirmed that the country's drug authority did not provide a medicine called migalastat, which is used in Scotland to treat the disease.

ELFABRIO is a PEGylated α-Gal A enzyme replacement therapy (ERT) that is approved for the treatment of adults with Fabry disease in the ... developed de novo anti-drug antibodies (ADAs) or ...

U.S. Food and Drug Administration (FDA) provides a clear regulatory ... its wholly owned gene therapy product candidate for the treatment of Fabry disease. The FDA has agreed in a Type B interaction ...