UniQure pauses dosing in Fabry disease trial amid safety concerns

Administration in the mid-to-high dose cohorts of the gene therapy trial will pause pending further evaluation.

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 ...

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 ...
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 ...

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 ...

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 ...

Aro Biotherapeutics Reports Preliminary Efficacy for ABX1100, a Muscle-targeted GYS1 siRNA, in Patients with Late-Onset Pompe Disease (LOPD)

Aro Biotherapeutics, a clinical-stage biotechnology company developing potent and tissue-targeted short-interfering RNA (siRNA) medicines, today announced interim data from a phase 1b study of ABX1100 ...
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 ...

Sangamo advances Fabry gene therapy with pivotal STAAR data

Detailed price information for Sangamo Therapeutics (SGMO-Q) from The Globe and Mail including charting and trades.
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.
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 ...