Amicus Therapeutics has announced that the first patient has been dosed in their Phase 3 study of AT-GAA for patients with Pompe disease.
Amicus Therapeutics announces positive data from their Phase 1/2 clinical study (ATB200-02) to investigate AT-GAA in both ERT-Naive and ERT-Switch patients with Pompe disease.
CRANBURY, N.J., Oct. 04, 2017 (GLOBE NEWSWIRE) — Amicus Therapeutics (Nasdaq:FOLD) today announced additional positive results from a global Phase 1/2 clinical study (ATB200-02) to investigate ATB200/AT2221 in patients with Pompe disease, an inherited lysosomal storage disorder caused by an enzyme deficiency that leads to accumulation of glycogen (disease substrate) in cells.
CRANBURY, N.J., (May 15, 2017) (GLOBE NEWSWIRE) – Amicus Therapeutics (Nasdaq:FOLD) today announced positive functional data from initial patients in a global Phase 1/2 study (ATB200-02) to investigate ATB200/AT2221 in patients with Pompe disease. Patients who completed six months of treatment with ATB200/AT2221 showed improvements in the six-minute walk test (6MWT) distance and other measures of motor function, in addition to stability or improvements in forced vital capacity (FVC).
CRANBURY, N.J. and SAN DIEGO, Feb. 15, 2017 (GLOBE NEWSWIRE) — Amicus Therapeutics (Nasdaq:FOLD), a global biotechnology company at the forefront of rare and orphan diseases, today presented new scientific findings and preclinical data on functional outcomes in an oral presentation and poster1 at the 13thAnnual WORLDSymposium™ in San Diego, CA. ATB200/AT2221 is a novel treatment paradigm that consists of ATB200, a unique recombinant human acid alpha-glucosidase (rhGAA) enzyme with optimized carbohydrate structures, particularly mannose-6 phosphate (M6P), to enhance uptake in muscles, co-administered with AT2221, a pharmacological chaperone designed to stabilize ERT in circulation.
CRANBURY, N.J. and SAN DIEGO, Feb. 15, 2017 (GLOBE NEWSWIRE) — Amicus Therapeutics (Nasdaq:FOLD), a global biotechnology company at the forefront of rare and orphan diseases, today presented additional positive preliminary data from a global Phase 1/2 study (ATB200-02) to investigate ATB200/AT2221 in a poster1 at the 13th Annual WORLDSymposium™ in San Diego, CA. ATB200/AT2221 is a novel treatment paradigm that consists of ATB200, a unique recombinant human acid alpha-glucosidase (rhGAA) enzyme with optimized carbohydrate structures, particularly mannose-6 phosphate (M6P), to enhance uptake, co-administered with AT2221, a pharmacological chaperone.
CRANBURY, N.J., Dec. 08, 2016 (GLOBE NEWSWIRE) — Amicus Therapeutics (Nasdaq:FOLD), a global biotechnology company at the forefront of rare and orphan diseases, today announced positive preliminary data from a global Phase 1/2 study (ATB200-02) to investigate ATB200/AT2221>. ATB200/AT2221 is a novel treatment paradigm that consists of ATB200, a unique recombinant human acid alpha-glucosidase (rhGAA) enzyme with optimized carbohydrate structures, particularly mannose-6 phosphate (M6P), to enhance uptake, co-administered with AT2221, a pharmacological chaperone.
Amicus Therapeutics (Nasdaq:FOLD), a biopharmaceutical company at the forefront of rare and orphan diseases, today announced that its investigational new drug (IND) application, submitted to U.S. Food and Drug Administration (FDA), is now effective which allows Amicus to begin site initiation and enrollment of a Phase 1/2 study of ATB200 in patients with Pompe disease.
CRANBURY, N.J., and ORLANDO, Fla., Feb. 14, 2013 (GLOBE NEWSWIRE) — Amicus Therapeutics (Nasdaq:FOLD) today announced positive results from clinical and preclinical studies of the pharmacological chaperone AT2220 (duvoglustat HCl) in combination with ERT for Pompe disease at the Lysosomal Disease Network WORLD Symposium.
CRANBURY, N.J., June 26, 2012 (GLOBE NEWSWIRE) — Amicus Therapeutics (Nasdaq:FOLD), today announced positive preliminary results from an ongoing Phase 2 open-label drug-drug interaction study (Study 010) to evaluate the safety and pharmacokinetic (PK) effects of the pharmacological chaperone AT2220 (duvoglustat HCl) co-administered with enzyme replacement therapy (ERT) for Pompe disease.