A nonprofit aimed at improving early diagnosis for a rare and life-threatening disorder is hosting its fifth annual fundraiser in the Upstate. Testing for Tots will host its 5th Annual Tasting for ...

The University of Alabama at Birmingham has special expertise in the evaluation and management of Rare Genetic Kidney disease. This clinic started due to a specific Rare Genetic Kidney disease called ...

This acceptance follows Sangamo’s meeting with the FDA in October 2025 to discuss the proposed efficacy and safety data package for isaralgagene civaparvovec where, in the meeting minutes, among other ...

A 29-year-old Fabry patient finds relief with a specific cannabis blend, reducing excruciating pain within a month. Medical cannabis offers hope for Fabry disease patients with severe neuropathic pain ...

Sangamo Therapeutics’ investigational gene therapy appears to improve kidney function at 52 weeks for patients with a rare genetic disorder. The biotech plans to use the data for an FDA submission ...

Shares of Sangamo Therapeutics (NASDAQ:SGMO) traded higher in the premarket on Tuesday after the company announced positive initial results from its pivotal Phase 1/2 STAAR study for its gene therapy ...

4D Molecular Therapeutics has offered to add an immunosuppressive regimen during the administration of a Fabry disease gene therapy in order to continue with human testing and lift a clinical hold ...

Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine company, today announced positive topline results from the registrational Phase 1/2 STAAR study evaluating isaralgagene civaparvovec, or ...

- U.S. Food and Drug Administration (FDA) provides a clear regulatory pathway to Accelerated Approval for isaralgagene civaparvovec using data from ongoing Phase 1/2 STAAR study, avoiding requirement ...

Fabrazyme (agalsidase beta) is a prescription drug that’s used to treat Fabry disease. The drug is given as an intravenous (IV) infusion by a healthcare professional. You’ll typically receive a dose ...