Fabry disease

News

Sangamo cues up Fabry gene therapy filing on STAAR data

Sangamo plans to file for approval of its gene therapy for Fabry disease next year, but a launch could depend on finding a commercial partner.

Digital

Medicine doctor writing electronic medical record

Driving innovation in rare diseases: Digital health and coll...

In recognition of Rare Disease Day, Dr Kamran Iqbal, head of medical affairs and patient advocacy in global rare diseases at Chiesi UK, discussed with pharmaphorum the company’s evolving ro

News

4DMT's Fabry gene therapy on hold, but 'remains active'

4D Molecular Therapeutics (4DMT) has said a trial of its gene therapy for Fabry disease has been placed on a clinical hold by the FDA, a few weeks after it reported three

News

Switch data back Chiesi/Protalix long-acting Fabry drug

Chiesi and Protalix have built the case for their long-acting enzyme replacement therapy (ERT) for Fabry disease PRX-102, as the FDA’s review of the drug continues towards a decision due by

News

Chiesi’s flagship Fabry drug heads for FDA verdict in early ...

The FDA has started its review of Israeli biotech Protalix BioTherapeutics and partner Chiesi’s Fabry disease therapy pegunigalsidase alfa, setting up a possible approval by 27

News

Chiesi starts rare diseases division, ahead of potential Fab...

Italian pharma Chiesi has begun a new rare diseases division as it builds towards a potential commercial launch for its Fabry Disease therapy.

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