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 ...

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.

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 ...

We recently published a list of 10 Best Low Priced Biotech Stocks to Buy Now. In this article, we are going to take a look at ...

The questionnaire contained questions on general health and the presence or absence of manifestations of the disease as is seen in affected ... which included “Fabry rash” (angiokeratoma), sweating, ...

Background Patients with Fabry disease (FD) show left ventricular hypertrophy (LVH) mimicking hypertrophic cardiomyopathy (HCM) of sarcomeric origin and might benefit, if detected early, from specific ...