Collaborations Pharmaceuticals, Inc. demonstrates a treatment for CLN1 Batten disease crosses the blood-brain-barrier

PPT1 is a recombinant protein used to treat an ultra-rare disease called Batten disease CLN1 for which there is ...
MPR

Venglustat Improves Neurological Symptoms in Type 3 Gaucher Disease

Findings from the LEAP2MONO study support the regulatory submission of venglustat for treatment of type 3 Gaucher disease.

Press Release: Sanofi’s venglustat met all primary endpoints in a phase 3 study of type 3 Gaucher disease

Sanofi’s venglustat met all primary endpoints in a phase 3 study of type 3 Gaucher diseaseIn the LEAP2MONO phase 3 study, venglustat, dosed ...

Sanofi's Venglustat Meets Primary Endpoints in Gaucher Disease Study

SNY's phase III LEAP2MONO study meets all primary endpoints, with venglustat improving neurological symptoms in type 3 ...

Protalix says EU panel recommended a new dosing regimen for Fabry disease therapy

Protalix BioTherapeutics (PLX) said on Friday that an expert panel of the European Medicines Agency (EMA), endorsed a new ...
MarketWatch

Spruce Biosciences Receives U.S. FDA Breakthrough Therapy Designation for Tralesinidase Alfa Enzyme Replacement Therapy (TA-ERT) in Sanfilippo Syndrome Type B (MPS IIIB)

Spruce Biosciences, Inc. (Nasdaq: SPRB), a late-stage biopharmaceutical company focused on developing and commercializing novel therapies for neurological disorders with significant unmet medical need ...

Sanofi's genetic disorder drug shows mixed results in late-stage trials

By Bhanvi Satija LONDON, Feb 2 (Reuters) - French drugmaker Sanofi said on Monday that its experimental genetic disorder treatment showed promise in a late-stage study of patients with a type of ...
Healio

Enzyme-replacement therapy improves outcomes in Pompe disease

Please provide your email address to receive an email when new articles are posted on . Researchers analyzed 19 children with Pompe disease seen at a Texas-based children’s hospital.
Medical Xpress

Therapeutic or Preventive Procedure:

A study evaluating a pioneering lentivirus (LV)-mediated gene therapy trial for classical Fabry disease showed promising results over five years, indicating a potential breakthrough in treatment for ...

Spur Therapeutics Presents New Phase 1/2 Data on Its Gene Therapy Candidate in Gaucher Disease at 22nd Annual WORLDSymposium™

Two-year follow-up data demonstrate sustained benefit, including improvements in pain, fatigue and bone health, following single low dose of avigbagene parvec (FLT201) Robust improvements in lyso-Gb1, ...
Business Insider

Dyne Therapeutics Presents New Preclinical Data Demonstrating the Potential of the FORCE™ Platform to Deliver Enzyme Replacement Therapy to Muscle and CNS in Pompe Disease

WALTHAM, Mass., June 24, 2024 (GLOBE NEWSWIRE) -- Dyne Therapeutics, Inc. (Nasdaq: DYN), a clinical-stage muscle disease company focused on advancing innovative life-transforming therapeutics for ...
WRAL

Enzyme Replacement Therapy Being Used To Treat Hunter Syndrome

Children with Hunter Syndrome lack a certain enzyme in their blood. The Food and Drug Administration just approved an enzyme-replacement therapy for these patients. It had its start at UNC Hospitals.