Acid alpha-glucosidase

Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) - 2021 Drugs in Development Report - ResearchAndMarkets.com

Retrieved on: 
Friday, June 4, 2021
Health, Pharmaceutical, Enzymes, Lysosomal storage diseases, Maltase, Acid alpha-glucosidase, Alpha-glucosidase, Hydrolases, Carbohydrates, Lysosome, Glucosidases, Glucocerebrosidase, GAA, Glucan 1,4-a-glucosidase

The "Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) - Drugs in Development, 2021" drug pipelines has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) - Drugs in Development, 2021" drug pipelines has been added to ResearchAndMarkets.com's offering.
  • According to the recently published report Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) pipeline Target constitutes close to 19 molecules.
  • The report outlays comprehensive information on the Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) targeted therapeutics, complete with analysis by indications, stage of development, mechanism of action (MoA), route of administration (RoA) and molecule type; that are being developed by Companies / Universities.
  • It also reviews key players involved in Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) targeted therapeutics development with respective active and dormant or discontinued projects.

Amicus Therapeutics Announces Upcoming Presentations at the 16th International Congress on Neuromuscular Diseases

Retrieved on: 
Friday, May 21, 2021
Clinical medicine, Medicine, Health sciences, Orphan drugs, Rare diseases, Glycogen storage disease type II, Hepatology, Alglucosidase alfa, Amicus Therapeutics, Acid alpha-glucosidase, Pompe

b'PHILADELPHIA, May 21, 2021 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq: FOLD), today announced two posters for presentation highlighting its development program for Pompe disease at the 16th International Congress on Neuromuscular Diseases\xc2\xa0(ICNMD) being held virtually, May 21-22 and 28-29, 2021.\nEnhancing Delivery of Acid Alpha-Glucosidase to Skeletal Muscle in Pompe Disease: Key Challenges and Attributes of AT-GAA\nEfficacy and safety of cipaglucosidase alfa/miglustat versus alglucosidase alfa/placebo in late-onset Pompe disease: PROPEL study\nThe posters will be made available on the Amicus website following their respective presentations at the congress.\nFor more information on the 16th International Congress on Neuromuscular Diseases, please visit www.icnmd.org .\nAmicus Therapeutics (Nasdaq: FOLD) is a global, patient-dedicated biotechnology company focused on discovering, developing and delivering novel high-quality medicines for people living with rare metabolic diseases.

Key Points: 
  • b'PHILADELPHIA, May 21, 2021 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq: FOLD), today announced two posters for presentation highlighting its development program for Pompe disease at the 16th International Congress on Neuromuscular Diseases\xc2\xa0(ICNMD) being held virtually, May 21-22 and 28-29, 2021.\nEnhancing Delivery of Acid Alpha-Glucosidase to Skeletal Muscle in Pompe Disease: Key Challenges and Attributes of AT-GAA\nEfficacy and safety of cipaglucosidase alfa/miglustat versus alglucosidase alfa/placebo in late-onset Pompe disease: PROPEL study\nThe posters will be made available on the Amicus website following their respective presentations at the congress.\nFor more information on the 16th International Congress on Neuromuscular Diseases, please visit www.icnmd.org .\nAmicus Therapeutics (Nasdaq: FOLD) is a global, patient-dedicated biotechnology company focused on discovering, developing and delivering novel high-quality medicines for people living with rare metabolic diseases.
  • With extraordinary patient focus, Amicus Therapeutics is committed to advancing and expanding a robust pipeline of cutting-edge, first- or best-in-class medicines for rare metabolic diseases.
  • For more information please visit the company\xe2\x80\x99s website at www.amicusrx.com , and follow us on Twitter and LinkedIn .\n'

Amicus Therapeutics Announces Successful Pre-BLA Meeting with U.S. FDA for AT-GAA for the Treatment of Pompe Disease

Retrieved on: 
Monday, May 3, 2021
Acid alpha-glucosidase, Glycogen storage disease type II, Pompe, Amicus Therapeutics, Health sciences, Clinical medicine, Health

We are moving ahead expeditiously with our submission and all the work necessary to ensure that AT-GAA gets to as many people living with Pompe disease as quickly as possible.

Key Points: 
  • We are moving ahead expeditiously with our submission and all the work necessary to ensure that AT-GAA gets to as many people living with Pompe disease as quickly as possible.
  • "\nJeff Castelli, Ph.D., Chief Development Officer of Amicus Therapeutics, stated, "We believe the data from our clinical trials, including the largest pivotal study ever completed in Pompe disease, have shown clinically meaningful improvements.
  • There can be no assurance that the FDA will accept a BLA submission or if accepted will grant approval for AT-GAA.
  • You are cautioned not to place undue reliance on these forward-looking statements, which speak only of the date hereof.

Amicus’ AT-GAA Shows Clinically Meaningful & Significant Improvements in Both Musculoskeletal and Respiratory Measures in Late-Onset Pompe Disease Compared to Standard of Care in Pivotal Phase 3 PROPEL Study

Retrieved on: 
Thursday, February 11, 2021
Branches of biology, Clinical medicine, Medicine, Hepatology, Lysosomal storage diseases, Rare diseases, Exercise physiology, Inborn errors of carbohydrate metabolism, Glycogen storage disease type II, Alglucosidase alfa, Pompe, Acid alpha-glucosidase

PROMIS Fatigue: Fatigue as measured by this scale slightly favored AT-GAA treated patients over alglucosidase alfa treated patients.

Key Points: 
  • PROMIS Fatigue: Fatigue as measured by this scale slightly favored AT-GAA treated patients over alglucosidase alfa treated patients.
  • Urine Hex-4 is a common biomarker in Pompe disease and is used as an indirect measure of the degree of skeletal glycogen clearance in Pompe patients receiving ERT.
  • Glycogen is the substrate that accumulates in the lysosomes of muscles of Pompe patients.
  • We believe that AT-GAA has the potential to quickly become the new standard of care in the treatment of this devastating muscle disease.

Amicus Therapeutics Initiates Rolling Biologic License Application to the U.S. Food and Drug Administration for AT-GAA in Late-Onset Pompe Disease

Retrieved on: 
Tuesday, December 1, 2020
Amicus Therapeutics, Gene therapy, Health sciences, Glycogen storage disease type II, Health, Clinical medicine, Amicus, Acid alpha-glucosidase, John Crowley

The FDA earlier this year authorized Amicus to proceed with a Rolling BLA submission.

Key Points: 
  • The FDA earlier this year authorized Amicus to proceed with a Rolling BLA submission.
  • John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics, stated, This submission represents a major milestone for Amicus and the entire Pompe community.
  • Amicus is also conducting an ongoing global Phase 3 clinical study (ATB200-03, or PROPEL) of AT-GAA in adult patients with late onset Pompe disease (LOPD).
  • With extraordinary patient focus, Amicus Therapeutics is committed to advancing and expanding a robust pipeline of cutting-edge, first- or best-in-class medicines for rare metabolic diseases.

Amicus Therapeutics Announces Upcoming Presentations at the American Society of Gene & Cell Therapy 23rd Annual Meeting

Retrieved on: 
Tuesday, May 5, 2020
Branches of biology, Medicine, Inborn errors of carbohydrate metabolism, Biology, Rare diseases, Hepatology, Lysosomal storage diseases, Glycogen storage disease type II, Acid alpha-glucosidase, Pompe, Gene therapy, Weakness

Preclinical data from its Pompe gene therapy program, which Amicus is developing with the Gene Therapy Program of the Perelman School of Medicine at the University of Pennsylvania, will be presented as an oral presentation.

Key Points: 
  • Preclinical data from its Pompe gene therapy program, which Amicus is developing with the Gene Therapy Program of the Perelman School of Medicine at the University of Pennsylvania, will be presented as an oral presentation.
  • Reduced or absent levels of GAA leads to accumulation of glycogen in cells, which results in the clinical manifestations of Pompe disease.
  • The disease can be debilitating and is characterized by severe muscle weakness that worsens over time.
  • You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof.

Muscular Dystrophy Association to host first Patient Focused Drug Development Meeting for Pompe Disease March 9, 2020 in Washington D.C.

Retrieved on: 
Thursday, November 21, 2019
Health, Medicine, Inborn errors of carbohydrate metabolism, Glycogen storage disease type II, Hepatology, Medical specialties, Pompe, Acid alpha-glucosidase, Muscular Dystrophy Association, MDA, Food and Drug Administration, Dystrophy

NEW YORK, Nov. 21, 2019 /PRNewswire/ --The Muscular Dystrophy Association (MDA) announced today that the U.S. Food and Drug Administration (FDA) has approved its externally-led Patient Focused Drug Development (PFDD) meeting on Pompe disease on March 9, 2020 in Hyattsville, Maryland.

Key Points: 
  • NEW YORK, Nov. 21, 2019 /PRNewswire/ --The Muscular Dystrophy Association (MDA) announced today that the U.S. Food and Drug Administration (FDA) has approved its externally-led Patient Focused Drug Development (PFDD) meeting on Pompe disease on March 9, 2020 in Hyattsville, Maryland.
  • MDA is partnering with the Acid Maltase Deficiency Association (AMDA) and United Pompe Foundation (UPF) to bring the Pompe community to Washington, D.C. in person and online.
  • This input will help inform FDA's regulatory decision making and oversight during therapy development and in the regulatory review process.
  • This report will provide lasting lessons on life with Pompe disease and the community's viewpoints on potential therapeutic interventions.

Danon Disease Pipeline Insight Report 2019: Glycogen Storage Disease Type 2B, Glycogen Storage Disease Type IIb, Lysosomal Glycogen Storage Disease with Normal Acid Maltase - ResearchAndMarkets.com

Retrieved on: 
Tuesday, September 17, 2019
Health, Pharmaceutical, Branches of biology, Rare diseases, Hepatology, Medical specialties, Health, Inborn errors of carbohydrate metabolism, Danon disease, Acid alpha-glucosidase, Metabolism, Glycogen storage disease type II, Glycogen storage disease, Glycogen

Danon Disease - Pipeline Insight, 2019 report outlays comprehensive insights of present scenario and growth prospects across the indication.

Key Points: 
  • Danon Disease - Pipeline Insight, 2019 report outlays comprehensive insights of present scenario and growth prospects across the indication.
  • A detailed picture of the Danon Disease pipeline landscape is provided which includes the disease overview and Danon Disease treatment guidelines.
  • The assessment part of the report embraces, in depth Danon Disease commercial assessment and clinical assessment of the pipeline products under development.
  • Danon Disease (Glycogen Storage Disease Type 2B, Glycogen Storage Disease Type IIb, Lysosomal Glycogen Storage Disease with Normal Acid Maltase)

AskBio Announces First Patient Dosed with Gene Therapy in Phase 1/2 Study of ACTUS-101 in Patients with Pompe Disease

Retrieved on: 
Tuesday, January 29, 2019
Branches of biology, Articles, Biology, Gene delivery, Medical genetics, Rare diseases, Applied genetics, Molecular biology, Glycogen storage disease type II, Acid alpha-glucosidase, Therapy, Pompe

Pompe Disease is a serious disease associated with high morbidity and often leads to premature death.

Key Points: 
  • Pompe Disease is a serious disease associated with high morbidity and often leads to premature death.
  • "ACTUS-101 is a next generation gene therapy, reflective of our commitment to discovering, developing, and manufacturing innovative AAV gene therapy candidates."
  • Dr. Koeberl stated, "Preclinical data suggest that this gene therapy product may prove to continuously produce the GAA they (Pompe patients) are lacking.
  • Actus-101 is an AAV gene therapy treatment for Pompe Disease now in a Phase 1/2 clinical study.