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 ...
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.
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 ...

uniQure Announces Updated Preliminary AMT-191 Phase I/IIa Data Showing Sustained Increases in α-Gal A Enzyme Activity in Patients with Fabry Disease

Supraphysiological expression of α-Gal A activity maintained for over a year in longest treated patient as of data cutoff date ~~ Stable ...
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 ...

Early UniQure Fabry trial shows lasting enzyme boost, safety watch

uniQure N.V. QURE on Friday announced updated preliminary safety and exploratory efficacy data from 11 patients in its Phase ...
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 ...