Healio

Enzyme replacement therapy curbs bleeding events in congenital blood clotting disorder

Please provide your email address to receive an email when new articles are posted on . Mean maximum ADAMTS13 activity after recombinant ADAMTS13 exceeded 100%. Markedly fewer patients receiving ...
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 ...
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.
Stocktwits on MSN

SPRB stock drops after company pushes back application submission timeline for experimental Sanfilippo syndrome therapy

The company had previously said that it expects to submit an application for approval of the therapy in the first quarter of 2026. ・In the December meeting with the FDA, the company discussed its ...
Yahoo Finance

Long-Term Data Presented at the 22nd Annual WORLDSymposium™ Highlights Tralesinidase Alfa Enzyme Replacement Therapy’s Potential as the First Disease-Modifying Treatment ...

Treatment with Weekly TA-ERT Demonstrated Rapid and Durable Normalization of Cerebral Spinal Fluid Heparan Sulfate Non-Reducing End (CSF HS-NRE), a Surrogate Endpoint Reasonably Likely to Predict ...
InvestorsHub on MSN

Spruce Biosciences reports positive FDA meetings as it prepares TA-ERT filing for rare pediatric disorder

Spruce Biosciences, Inc. (NASDAQ:SPRB) said it has completed two Type B meetings with the U.S. Food and Drug Administration (FDA), clearing key regulatory discussions ahead of its planned biologics ...
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 ...
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 ...
Medical Xpress

Therapeutics: News and Research on Enzyme Therapy

Ongoing clinical research at UNC could lead to a first-of-its-kind enzyme replacement therapy for Hunter syndrome, an ultra-rare disorder that causes progressive multisystem disease and neurologic ...
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% ...
Seeking Alpha

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

Breakthrough Therapy Designation Supported by Integrated Long-Term Clinical Data Demonstrating Normalization in Cerebral Spinal Fluid Heparan Sulfate Non-Reducing End (CSF HS-NRE) U.S. FDA Confirmed ...