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BioWorld - Saturday, June 27, 2026
Home » enzyme replacement therapy

Articles Tagged with ''enzyme replacement therapy''

Alpha-galactosidase enzyme
Travel issues strike again

Protalix shares crumble on CRL for Fabry disease therapy

April 28, 2021
By Michael Fitzhugh
Shares of Protalix Biotherapeutics Inc. (NYSE:PLX) fell 33% to $3.93 on April 28 after the company reported receiving an FDA complete response letter (CRL) in answer to its BLA for pegunigalsidase alfa, a galactosidase enzyme replacement therapy it has long advanced for the potential treatment of Fabry disease.
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Limb exam

Amicus takes walk-test outcome in stride as Pompe BLA rolls on

Feb. 12, 2021
By Randy Osborne
Amicus Therapeutics Inc.’s results from the phase III trial called Propel with AT-GAA (cipaglucosidase alfa and miglustat) for late-onset Pompe disease (LOPD) met with split opinions, though Wall Street took a decidedly dim view and left shares (NASDAQ:FOLD) to close at $12.57, down $6.16, or 33%.
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M&A cityscape

Not-little Orphan ante, more HCU Warbucks ahead in $517M Retrophin deal

Oct. 23, 2020
By Randy Osborne
CEO Eric Dube said Retrophin Inc. will “share more [about clinical development plans] once the deal has closed” in the fourth quarter of this year and Orphan Technologies Ltd. belongs to his firm, which has seen only animal data so far with OT-58, an enzyme replacement therapy (ERT) for classical homocystinuria (HCU).
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