Amicus Therapeutics Receives Breakthrough Therapy Designation for Pompe Treatment
Amicus Therapeutics announced today that they have received Breakthrough Therapy Designation (BTD) for their AT-GAA treatment for Pompe disease.
Amicus Therapeutics Announces First Patient Dosed in Phase 3 PROPEL Study of AT-GAA in Patients with Pompe Disease
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 18-Month Data in Pompe Disease Phase 1/2 Study
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.
Amicus Therapeutics Announces Additional Positive Data in Pompe Disease Phase 1/2 Study at World Muscle Society
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.
Amicus Therapeutics Announces Positive Functional Data from Initial Patients in Pompe Phase 1/2 Study
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).
Amicus Therapeutics Presents Important New Scientific Findings and Preclinical Data for Pompe Program at WORLDSymposium™ 2017
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.
Amicus Therapeutics Presents Additional Positive Preliminary Phase 1/2 Data at WORLDSymposium™ 2017
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.
Amicus Therapeutics Announces Positive Preliminary Data from Phase 1/2 Study of Novel Treatment Paradigm for Pompe Disease
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 Commences Phase 1/2 Study of Novel ERT for Treatment of Pompe Disease
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.
Amicus Therapeutics Advances Chaperone-Enzyme Replacement Therapy (ERT) Combination Platform in 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.