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 ...

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 ...

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 ...
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.
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 ...
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 ...

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 ...
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.
MedPage Today

Gene Therapy Shows Lasting Benefit for Children With Rare Disorder

An investigational gene therapy showed sustained clinical efficacy for children born with ADA-SCID. In a study of 62 children, overall survival after treatment was 100% and event-free survival was 95% ...