Sangamo Therapeutics Inc (SGMO) reports significant milestones and strategic initiatives, despite financial challenges, in ...

Announced derisking milestones in pathway to anticipated biologics license application (BLA) submission for isaralgagene civaparvovec in Fabry disease, including all patients having passed one-year ...

The STAAR study is a global, open-label, single-dose, dose-ranging trial designed to assess the safety and tolerability of ST-920 in patients with Fabry disease, a lysosomal storage disorder ...

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.

Air Force veteran Melissa Hay battled years of misdiagnosis before discovering she had Fabry disease—a rare, life-threatening ...

Sangamo aims to submit a BLA for Fabry disease treatment in Q1 2026. Significant cost reductions were achieved, extending the cash runway to late Q3 2025. Sangamo Therapeutics is focusing on its ...

Chiesi GRD has an initial focus in developing therapies for lysosomal storage disorders (LSDs), including Fabry disease, as well as other rare diseases in haematology and opthalmology indications.

Amicus Therapeutics FOLD reported first-quarter 2025 adjusted earnings of 3 cents per share, which missed the Zacks Consensus Estimate of 8 cents. The company had reported a loss of 2 cents per share ...

This particular candidate is being developed to treat patients with Fabry Disease. A good update was also provided here a few months ago, whereby the independent data monitoring committee (IDMC ...

In the first quarter of 2025, the Company completed a public offering of 5.1 million ordinary shares, including the full exercise of the underwriters’ overallotment option, at a public offering price ...

Alexander Macrae; President, Chief Executive Officer, Director; Sangamo Therapeutics Inc Prathyusha Duraibabu; Chief Financial Officer, Senior Vice President; Sangamo Therapeutics Inc Good afternoon, ...

Azafaros has secured 132 million euros ($147 million) in series B funds that the Dutch biotech will use to push its rare ...