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 ...
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 ...

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

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 ...
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 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 ...
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.
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 ...
Fierce Biotech

UniQure pauses Fabry gene therapy dosing in 2 groups after toxicities emerge

Two dose-limiting toxicities have been recorded in uniQure’s trial evaluating an investigational AAV gene therapy designed to ...
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 ...