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- Fabry Disease

Fabry Disease Newsletter

Sangamo Therapeutics Initiates Rolling Submission of BLA to U.S. FDA for ST-920 in Fabry Disease 18 Dec 2025 13:05 GMT
… the treatment of adults with Fabry disease. Rolling submission allows for … time infusion without preconditioning. About Fabry Disease Fabry disease is a lysosomal storage disorder … time, durable treatment option for Fabry disease to provide meaningful, multi- …
BioMarin (BMRN) Jumps 17.7% on $5-Billion Merger with Amicus 20 Dec 2025 19:09 GMT
… Galafold, an oral treatment for Fabry disease, and Pombiliti + Opfolda, a two …
California biotech BioMarin will pay $4.8 billion for Amicus Therapeutics, a rare disease company with a presence in Philadelphia 20 Dec 2025 02:33 GMT
… Amicus has a treatment for Fabry disease, which is caused by a …
<![CDATA[BioMarin Reaches $4.8 Billion Definitive Agreement to Acquire Amicus Therapeutics ]]> 19 Dec 2025 23:08 GMT
… therapies for individuals living with Fabry disease and Pompe disease. BioMarin… , the first oral treatment for Fabry disease, and Pombiliti in combination with …
STAT+: BioMarin to buy Amicus Therapeutics for $4.8B, bringing together rare disease biotechs 19 Dec 2025 23:29 GMT
… Pompe disease and one for Fabry disease, that took in $600 million …
STAT+: BioMarin CEO says $4.8 billion Amicus deal will lead to cost savings and revenue growth 19 Dec 2025 23:28 GMT
… Pompe disease and one for Fabry disease, which took in $600 million …
BioMarin to buy rare disease drugmaker Amicus for $4.8B 19 Dec 2025 20:17 GMT
… , Galafold, is used to treat “Fabry disease,” a genetic, potentially life-threatening …
BioMarin’s Presence in Rare Enzyme Disorders Grows With $4.8B Amicus Therapeutics Acquisition 19 Dec 2025 20:11 GMT
… a different enzyme deficiency named Fabry disease. In 2023, Amicus won FDA …
BioMarin buys pharma peer Amicus for $4.8bn 19 Dec 2025 19:16 GMT
… 's Galafold drug for Fabry disease, and Pombiliti-Opfolda for Pompe … therapies for individuals living with Fabry disease and Pompe disease," said …
BioMarin makes its largest-ever transaction, paying $4.8B for fellow rare disease drugmaker Amicus 19 Dec 2025 18:29 GMT
… gains two rapidly growing products—Fabry disease drug Galafold and Pompe … growth potential with Galafold because Fabry disease is “a dramatically underdiagnosed condition … enzyme replacement franchise” into the Fabry disease and Pompe disease markets. It …

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