Healthline

What to Know About Gaucher Disease Type 1

Gaucher disease is a rare genetic disorder that causes the buildup of fatty substances in certain organs. Type 1 is the most common type of Gaucher disease, but it’s the least severe and has the most ...
Business Wire

AVROBIO Announces New Positive Clinical Data and Outlines Clinical Development Plan Following Regulatory Discussions for its Gaucher Disease Gene Therapy

New compelling clinical data from first-ever Gaucher disease type 3 (GD3) patient and four Gaucher disease type 1 (GD1) patients dosed with investigational AVR-RD-02 Data from first pediatric GD3 ...
Business Insider

Spur Therapeutics Showcases Positive New Data from Phase 1/2 GALILEO-1 Trial of FLT201, Its Novel Gene Therapy Candidate for Gaucher Disease, in Oral Presentation at ESGCT 31st ...

Data highlight potential of FLT201 to set new standard of care for Gaucher disease Dramatic and durable reductions observed in lyso-Gb1, an established biomarker of clinical response, in patients with ...
The Forward

New hope for Gaucher disease as experimental treatment begins trials

A new treatment may be on the horizon for a rare genetic disease that disproportionately affects Ashkenazi Jews. For years, the most common treatment for Type 1 was enzyme replacement therapy, but ...
BioWorld

Murine model recapitulates human Gaucher disease type 1

Researchers from the Yale University School of Medicine have developed a novel murine model of Gaucher disease type I with the aim to investigate the impact of GBA1 deficiency on hematopoiesis and the ...
FiercePharma

Experimental gene therapies on the cusp to ‘disrupt’ Gaucher disease drug market: GlobalData

As gene therapies ramp up in spinal muscular atrophy and hemophilia, it may soon be the turn of Gaucher disease patients to realize the latent potential of this type of treatment. That’s according to ...
Business Insider

Spur Therapeutics Presents Updated Phase 1/2 Data on Its Gene Therapy Candidate in Gaucher Disease at ESGCT 32nd Annual Congress

Single dose of avigbagene parvec (FLT201) demonstrates sustained clinical benefit and favorable safety up to two years after withdrawal of prior chronic therapy Data underscore potential to set new ...
Business Wire

AVROBIO Receives Rare Pediatric Disease Designation from the U.S. FDA for First-in-Class Gene Therapy for Gaucher Disease

CAMBRIDGE, Mass.--(BUSINESS WIRE)--AVROBIO, Inc. (Nasdaq: AVRO), a leading clinical-stage gene therapy company working to free people from a lifetime of genetic disease, today announced that the U.S.
The Forward

This Jewish disease wreaks havoc. But it may have an upside.

A rare genetic disorder that disproportionately affects Ashkenazi Jews is also a safeguard against tuberculosis, a new study shows, and that discovery could explain a longstanding scientific mystery.