Pompe

Maze Therapeutics Announces New Clinical Data Supporting MZE001 as a Potential Treatment for Pompe Disease

Retrieved on: 
Wednesday, March 22, 2023
Software, Research, General Health, Pharmaceutical, Oncology, Data Management, Technology, Genetics, Clinical Trials, Science, Biotechnology, Health, Tissue, Muscular Dystrophy Association, Pompe, Glycogen, Glycogen storage disease type II, Enzyme replacement therapy, Blood, HIV disease progression rates, Patient, Episcopal conference, MDA, Therapy, Mass drug administration, Conference, Biopsy, Pharmaceutical industry, Maze, PBMC

The data are being presented today, March 22, 2023, from 3:00-3:15 p.m. CT, during a late-breaking clinical session at the Muscular Dystrophy Association (MDA) Clinical and Scientific Conference in Dallas.

Key Points: 
  • The data are being presented today, March 22, 2023, from 3:00-3:15 p.m. CT, during a late-breaking clinical session at the Muscular Dystrophy Association (MDA) Clinical and Scientific Conference in Dallas.
  • The buildup of glycogen in skeletal, respiratory and cardiac muscle tissues is the primary driver of disease progression in patients with Pompe disease.
  • MZE001, a selective inhibitor of glycogen synthase 1 in muscle, is being developed to address this therapeutic gap.
  • Maze has developed a novel biomarker, peripheral blood mononuclear cell (PBMC) glycogen, as a surrogate for glycogen in skeletal muscle.

Amicus Therapeutics Announces Presentations and Posters at the 19th Annual WORLDSymposium™ 2023

Retrieved on: 
Monday, February 13, 2023
DO, Meta-analysis, Amicus Therapeutics, Glycogen storage disease type II, III, Diagnosis, Safety, Pompe, Patient, Hospital, University, Quality of life, Poster, Therapy, Fabry disease, Nursing, Pharmaceutical industry, Medical imaging

PHILADELPHIA, Feb. 13, 2023 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq: FOLD), today announced that two oral presentations and 11 posters across its development programs will be included at the 19th Annual WORLDSymposium™ 2023 , being held February 22-26, 2023 in Orlando, FL.

Key Points: 
  • PHILADELPHIA, Feb. 13, 2023 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq: FOLD), today announced that two oral presentations and 11 posters across its development programs will be included at the 19th Annual WORLDSymposium™ 2023 , being held February 22-26, 2023 in Orlando, FL.
  • Abstract Title: Long-term follow-up of cipaglucosidase alfa/miglustat in ambulatory patients with Pompe disease: An open-label phase I/II study (ATB200-02)
    Abstract Title: Long-term efficacy and safety of cipaglucosidase alfa/miglustat in ambulatory patients with Pompe disease: A phase III open-label extension study (ATB200-07)
    Presenter: Staci Kallish, DO, Hospital of the University of Pennsylvania, Philadelphia, PA, U.S.A.
    Abstract Title: FollowME Fabry Pathfinders registry: Renal effectiveness in a multi-national, multi-center cohort of patients on migalastat treatment for at least three years (Poster #176)
    Abstract Title: Gender heterogeneity in the diagnosis and treatment journey for patients with Fabry disease: A Japanese database analysis (Poster #81)
    Abstract Title: Long-term follow-up of cipaglucosidase alfa/miglustat in ambulatory patients with Pompe disease: An open-label phase I/II study (ATB200-02) (Poster #59)
    Abstract Title Long-term efficacy and safety of cipaglucosidase alfa/miglustat in ambulatory patients with Pompe disease: A phase III open-label extension study (ATB200-07): (Poster #LB-59)
    Abstract Title: Disease burden, treatment patterns and healthcare resource utilization associated with Pompe disease in Sweden: A real-world evidence study (Poster #257)
    Abstract Title: Indirect treatment comparison of three enzyme replacement treatments for late-onset Pompe disease: A network meta-analysis with patient-level and aggregate data (Poster #63)
    Abstract Title: Quality of life with late-onset Pompe disease: Qualitative interviews and general public utility estimation (Poster #236)

John F. Crowley, Executive Chairman and Founding CEO of Amicus Therapeutics, to Receive 2023 Horatio Alger Award

Retrieved on: 
Wednesday, January 18, 2023
News, American Dream, Extraordinary Measures, Foreign Service, United States service academies, University, Education, Therapy, Learning, Horatio Alger Association of Distinguished Americans, Biotechnology, Notre Dame, Family, Terrorism, Amicus Therapeutics, Georgetown University, JD, Love, Diagnosis, Triple accreditation, Intelligence, World War II, Glycogen storage disease type II, Pressure of speech, Member, Grandparent, Amicus, Harvard Business School, MBA, Horatio Alger, Pompe, Dream, Life, Perseverance, Pharmaceutical industry, Medicine

WASHINGTON, Jan. 18, 2023 /PRNewswire/ -- Horatio Alger Association of Distinguished Americans, Inc., a nonprofit educational organization honoring the achievements of outstanding individuals and encouraging youth to pursue their dreams through higher education, today announced that John F. Crowley, executive chairman and founding CEO of Amicus Therapeutics, has been selected for membership in this prestigious organization. Mr. Crowley joins 12 other exceptional business, civic and cultural leaders from across North America in receiving 2023 honors. For 76 years, the Horatio Alger Award has been annually bestowed upon esteemed individuals who have succeeded despite facing adversities, and who have remained committed to education and charitable efforts in their communities. 

Key Points: 
  • "His ability to persevere and find purpose through multiple adversities demonstrates what it means to be a Horatio Alger Member.
  • "Each year, it is our utmost honor to present outstanding leaders who have displayed extraordinary perseverance in overcoming challenges with the Horatio Alger Award," said James F. Dicke II, chairman, Horatio Alger Association and 2015 Horatio Alger Award recipient.
  • Mr. Crowley and the Member Class of 2023 will be formally inducted into the Association during the Horatio Alger Award Induction Ceremonies in Washington, D.C. from March 30 through April 1, 2023.
  • For more information about Horatio Alger Association and its Member Class of 2023, please visit www.horatioalger.org and follow the organization on Facebook , Twitter and Instagram .

Recombinant Adeno-Associated Virus (rAAV) Technology Pioneered by AskBio's Dr. Jude Samulski is Key Component of All FDA Approved AAV Gene Therapeutics

Retrieved on: 
Tuesday, December 20, 2022
Central nervous system, Cell, CSL, Glycogen storage disease type II, PARK, Novartis, Haemophilia, Molecular medicine, Spark Therapeutics, Duchenne, Research Triangle Park, AAV2, Capsid, Duchenne muscular dystrophy, FDA, AAV, Multimedia, Pompe, Therapy, Heart, Parkinson's disease, RESEARCH, Doctor of Philosophy, Kaay Raav Tumhi, Pharmaceutical industry, Kerosene, Bayer, Research

"The progress made in gene therapy to date is remarkable," said Jude Samulski, AskBio Co-Founder, President and Chief Scientific Officer.

Key Points: 
  • "The progress made in gene therapy to date is remarkable," said Jude Samulski, AskBio Co-Founder, President and Chief Scientific Officer.
  • My ultimate vision, shared by all of us at AskBio, is to make gene therapy more accessible for everyone in need around the world.
  • Dr. Samulski led the UNC Gene Therapy Center from 1993 to 2016 and, in 2001, co-founded AskBio, where many advances in recombinant AAV (rAAV) and underlying AAV vector technology were, and are being, made.
  • Since 2017, three AAV gene therapies have been approved for use by the FDA.

Maze Therapeutics Announces Completion of Phase 1 First-in-Human Trial Evaluating MZE001 as a Potential Oral Treatment for Pompe Disease

Retrieved on: 
Thursday, December 15, 2022
Health, Genetics, Clinical Trials, Pharmaceutical, Cardiology, Biotechnology, GAA, Clinical trial, PD, Glycogen storage disease type II, Data science, Cardiac muscle, Safety, Pharmacokinetics, Pompe, Glycogen, Bridge, Gene, Research, HIV disease progression rates, PK, Therapy, Compass, Tissue, Patient, Pharmaceutical industry, Maze

MZE001, an oral glycogen synthase (GYS1) inhibitor that aims to address Pompe disease by limiting disease-causing glycogen buildup, is being evaluated for the potential oral treatment of patients with Pompe disease.

Key Points: 
  • MZE001, an oral glycogen synthase (GYS1) inhibitor that aims to address Pompe disease by limiting disease-causing glycogen buildup, is being evaluated for the potential oral treatment of patients with Pompe disease.
  • Based on these findings, Maze plans to initiate a Phase 2 clinical trial of MZE001 in 2023.
  • We look forward to carrying this program forward into a Phase 2 clinical trial in patients with Pompe disease who need improved treatment options for their disease.
  • MZE001 is currently being evaluated for the potential oral treatment of patients with Pompe disease and potentially other glycogen storage disorders.

CANbridge Pharmaceuticals Granted Orphan Drug Designation for CAN 106 for the Treatment of Myasthenia Gravis

Retrieved on: 
Wednesday, November 16, 2022
Oncology, Health, FDA, General Health, Clinical Trials, Pharmaceutical, Biotechnology, FDA, Pompe, Weakness, Spinal muscular atrophy, Muscle contraction, Paroxysmal nocturnal hemoglobinuria, American Society of Gene and Cell Therapy, Acetylcholine, C5a, European Society for Medical Oncology, Glycogen storage disease, Ventilation, Glycogen storage disease type II, Neuromuscular disease, Degenerative disease, WuXi Biologics, University of Massachusetts, Patient, US, C3b, European Society of Gene and Cell Therapy, Muscle, Cell, C3a, SMA, Hospital, Glioblastoma, Research, MAC, Safety, Woman, Hunter syndrome, Drug, C3, Myasthenia gravis, Disease, Complement component 5, US FDA, Gene, C5, Congress, PNH, University, Haemophilia, Society, Fabry disease, Orphan, Pharmaceutical industry, Vaccine

Orphan Drug Designation for CAN106 in MG is both a validation of CANbridge innovation and a major milestone as our first US FDA regulatory designation, said James Xue, Ph.D., CANbridge Founder, Chairman and CEO.

Key Points: 
  • Orphan Drug Designation for CAN106 in MG is both a validation of CANbridge innovation and a major milestone as our first US FDA regulatory designation, said James Xue, Ph.D., CANbridge Founder, Chairman and CEO.
  • We continue to advance our global development strategy for CAN106 and look forward to developing CAN106 for myasthenia gravis and other complement-mediated diseases.
  • CAN106 is a clinical-stage investigational novel, long-acting recombinant humanized monoclonal antibody that binds to and neutralizes C5, a key component of the complement system.
  • CAN106 acts downstream of C3 in the complement pathway, preserving the generation of C3a and C3b, which are important for innate immunity.

Maze Therapeutics Appoints Harold Bernstein, M.D., Ph.D., as President, Research and Development and Chief Medical Officer

Retrieved on: 
Thursday, October 13, 2022
Health, Genetics, Other Health, Research, Science, Pharmaceutical, Biotechnology, Pompe, LinkedIn, Merck, CSO, Medicine, Glycogen storage disease type II, Therapy, Chronic kidney disease, Head, BioMarin Pharmaceutical, Science, Mount Sinai Kravis Children's Hospital, ALS Therapy Development Institute, Cardiology, Vertex, Harvard College, Patient, Twitter, Research, HIV disease progression rates, Internal medicine, History, Precision medicine, Technology, R, University, Industry, Mount Sinai School, Pediatrics, UCSF Benioff Children's Hospital, Icahn School of Medicine at Mount Sinai, Genetics, University of California, San Francisco, Stanford University, Third Rock Ventures, Bristol Myers Squibb, Systems biology, Gene, Data science, UCSF, Management, Pharmaceutical industry, Maze, Brigham, Compass

Maze Therapeutics, a company translating genetic insights into new precision medicines, today announced that Harold Bernstein, M.D., Ph.D., a 30-year industry veteran, has been appointed as president, head of research and development (R&D) and chief medical officer.

Key Points: 
  • Maze Therapeutics, a company translating genetic insights into new precision medicines, today announced that Harold Bernstein, M.D., Ph.D., a 30-year industry veteran, has been appointed as president, head of research and development (R&D) and chief medical officer.
  • In addition, Eric Green, M.D., Ph.D., who has served as Mazes senior vice president, research and translational sciences, has been promoted to chief scientific officer.
  • Dr. Bernstein brings more than three decades of experience in basic scientific research, translational medicine and clinical development both in industry and academia.
  • He joins Maze from BioMarin, where he served as senior vice president, chief medical officer and head of global clinical development.

Amicus Therapeutics Announces Participation at World Muscle Society 2022

Retrieved on: 
Tuesday, October 11, 2022
Pompe, LinkedIn, Congress, Glycogen storage disease type II, Therapy, Oldbury, Lists of diseases, Amicus Therapeutics, Patient, Poster, Glycogen storage disease, Twitter, Nasdaq, Degenerative disease, Association, Board, GLOBE, Meta-analysis, COVID-19, Pharmaceutical industry, Online gambling, Trustee

PHILADELPHIA, Oct. 11, 2022 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq: FOLD), today announced four poster presentations highlighting its development program for Pompe disease will be included at the 27th International Hybrid Annual Congress of the World Muscle Society , being held October 11-15, 2022 both virtually and in Halifax, Canada.

Key Points: 
  • PHILADELPHIA, Oct. 11, 2022 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq: FOLD), today announced four poster presentations highlighting its development program for Pompe disease will be included at the 27th International Hybrid Annual Congress of the World Muscle Society , being held October 11-15, 2022 both virtually and in Halifax, Canada.
  • For more information on the World Muscle Society 2022 Annual Congress, please visit www.wms2022.com .
  • Amicus 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 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.

Amicus Therapeutics Announces Participation at the 2022 AANEM Annual Meeting

Retrieved on: 
Wednesday, September 21, 2022
Lists of diseases, Glycogen storage disease type II, Twitter, Amicus Therapeutics, PROPEL, Annual general meeting, LinkedIn, Poster, American Association, Therapy, Electrodiagnostic medicine, AANEM, Patient, Pompe, Association, GLOBE, Pharmaceutical industry

PHILADELPHIA, Sept. 21, 2022 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq: FOLD), today announced three poster presentations highlighting its development program for Pompe disease will be included at the 2022 American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) Annual Meeting , being held September 21-24, 2022 in Nashville, TN and virtually.

Key Points: 
  • PHILADELPHIA, Sept. 21, 2022 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq: FOLD), today announced three poster presentations highlighting its development program for Pompe disease will be included at the 2022 American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) Annual Meeting , being held September 21-24, 2022 in Nashville, TN and virtually.
  • For more information on the 2022 AANEM Annual Meeting, please visit www.aanem.org .
  • Amicus 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.

Insights on the Enzyme Replacement Therapy Global Market to 2028 - Players Include Sanofi, BioMarin Pharmaceutical and Takeda Pharmaceutical - ResearchAndMarkets.com

Retrieved on: 
Monday, September 12, 2022
Biotechnology, Pharmaceutical, Genetics, Health, Enzyme replacement therapy, Enzyme, Fabry disease, Department, COVID-19, Hurler syndrome, Prevalence, City, Tay–Sachs disease, Administration, Growth, Glycogen storage disease type II, Infection, Protein, ERT, Glycogen storage disease, Gaucher's disease, European Medicines Agency, Pompe, Marketing, Mortality, LSD, Emergency medicine, EMA, 40, CAGR, Pharmaceutical industry

The "Enzyme Replacement Therapy Market Forecast to 2028 - COVID-19 Impact and Global Analysis By Enzyme Type, Therapeutic Conditions, Route of Administration, and End User" report has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "Enzyme Replacement Therapy Market Forecast to 2028 - COVID-19 Impact and Global Analysis By Enzyme Type, Therapeutic Conditions, Route of Administration, and End User" report has been added to ResearchAndMarkets.com's offering.
  • The enzyme replacement therapy market is expected to grow from US$ 9,673.39 million in 2021 to US$ 15,184.70 million by 2028; it is estimated to grow at a CAGR of 6.8% from 2022 to 2028.
  • The growth of the global enzyme replacement therapy market is attributed to the rising prevalence of lysosomal storage diseases (LSDs) and rapid regulatory approval with other marketing benefits for drug with orphan drug designation.
  • Enzyme replacement therapy (ERT) is performed to treat congenital enzyme deficiencies by using enzymes or proteins having enzymatic activity.