ERT

E-Clinical Solutions Market size worth USD 17.6 Billion, Globally, by 2030 at 14.3% CAGR: Verified Market Research®

Retrieved on: 
Friday, October 13, 2023
Investment, Geography, Clinical data management, Oracle Corporation, USD, Bank statement, Medidata Solutions, Cancer, Diabetes, Growth, AIDS, Parexel, ERT, Research, Dietary supplement, Pharmaceutical industry, Fine chemical

JERSEY CITY, N.J., Oct. 13, 2023 /PRNewswire/ -- The Global E-Clinical Solutions Market is projected to grow at a CAGR of 14.3% from 2023 to 2030, according to a new report published by Verified Market Research®. The report reveals that the market was valued at USD 6.90 Billion in 2022 and is expected to reach USD 17.6 Billion by the end of the forecast period.

Key Points: 
  • The " Global E-Clinical Solutions Market Size By Type, By Delivery Mode, By Geographic Scope And Forecast " report has been published by Verified Market Research®.
  • The report provides an in-depth analysis of the global E-Clinical Solutions Market, including its growth prospects, market trends, and market challenges.
  • To get market data, market insights, financial statements and a comprehensive analysis of the Global E-Clinical Solutions Market, please Contact Verified Market Research® .
  • Based on the research, Verified Market Research® has segmented the global E-Clinical Solutions Market into Type, Delivery Mode, And Geography.

WuXi Biologics Congratulates Amicus Therapeutics on U.S. FDA Approval for New Treatment for Pompe Disease

Retrieved on: 
Monday, October 2, 2023
Partnership, Therapy, Glycogen storage disease type II, Medicine, Development, Capsule, ERT, GAA, Contract research organization, Patient, WuXi Biologics, Rare, Amicus Therapeutics, Enzyme replacement therapy, FDA, Disease, Fine chemical, Pharmaceutical industry

WUXI, China, Oct. 1, 2023 /PRNewswire/ -- WuXi Biologics ("WuXi Bio") (2269.HK), a leading global Contract Research, Development and Manufacturing Organization (CRDMO), congratulates its strategic partner, Amicus Therapeutics ("Amicus") (Nasdaq: FOLD), on receiving U.S. FDA's approval for Pombiliti™ (cipaglucosidase alfa-atga) + Opfolda™ (miglustat) 65mg capsules.

Key Points: 
  • WUXI, China, Oct. 1, 2023 /PRNewswire/ -- WuXi Biologics ("WuXi Bio") (2269.HK), a leading global Contract Research, Development and Manufacturing Organization (CRDMO), congratulates its strategic partner, Amicus Therapeutics ("Amicus") (Nasdaq: FOLD), on receiving U.S. FDA's approval for Pombiliti™ (cipaglucosidase alfa-atga) + Opfolda™ (miglustat) 65mg capsules.
  • This two-component therapy is indicated for those adults living with late-onset Pompe disease (LOPD) weighing ≥40 kg and who are not improving on their current enzyme replacement therapy (ERT).
  • Bradley Campbell, President and Chief Executive Officer of Amicus Therapeutics, commented, "This approval marks a major milestone for the Pompe disease community in the United States.
  • Dr. Chris Chen, CEO of WuXi Biologics, commented, "We're honored to enable Amicus to achieve this great milestone, which we've been looking forward to for over 11 years.

WuXi Biologics Congratulates Amicus Therapeutics on U.S. FDA Approval for New Treatment for Pompe Disease

Retrieved on: 
Monday, October 2, 2023
Partnership, Therapy, Glycogen storage disease type II, Medicine, Development, Capsule, ERT, GAA, Contract research organization, Patient, WuXi Biologics, Rare, Amicus Therapeutics, Enzyme replacement therapy, FDA, Disease, Fine chemical, Pharmaceutical industry

WUXI, China, Oct. 2, 2023 /PRNewswire/ -- WuXi Biologics ("WuXi Bio") (2269.HK), a leading global Contract Research, Development and Manufacturing Organization (CRDMO), congratulates its strategic partner, Amicus Therapeutics ("Amicus") (Nasdaq: FOLD), on receiving U.S. FDA's approval for Pombiliti™ (cipaglucosidase alfa-atga) + Opfolda™ (miglustat) 65mg capsules.

Key Points: 
  • WUXI, China, Oct. 2, 2023 /PRNewswire/ -- WuXi Biologics ("WuXi Bio") (2269.HK), a leading global Contract Research, Development and Manufacturing Organization (CRDMO), congratulates its strategic partner, Amicus Therapeutics ("Amicus") (Nasdaq: FOLD), on receiving U.S. FDA's approval for Pombiliti™ (cipaglucosidase alfa-atga) + Opfolda™ (miglustat) 65mg capsules.
  • This two-component therapy is indicated for those adults living with late-onset Pompe disease (LOPD) weighing ≥40 kg and who are not improving on their current enzyme replacement therapy (ERT).
  • Bradley Campbell, President and Chief Executive Officer of Amicus Therapeutics, commented, "This approval marks a major milestone for the Pompe disease community in the United States.
  • Dr. Chris Chen, CEO of WuXi Biologics, commented, "We're honored to enable Amicus to achieve this great milestone, which we've been looking forward to for over 11 years.

JCR Pharmaceuticals Announces 52-Week Interim Data from its Global Phase I/II Study of JR-171 in Individuals with Mucopolysaccharidosis Type I (MPS I)

Retrieved on: 
Friday, September 29, 2023
Health, General Health, Clinical Trials, Research, Science, Pharmaceutical, Biotechnology, Communication, Central nervous system, DSM-IV codes, Biomarker, Cerebrospinal fluid, MPS, CNS, Trial of the century, BBB, MPS I, ERT, Patient, UCSF, INN, UCSF Benioff Children's Hospital, Children's Hospital Oakland Research Institute, TSE, Hurler, Pharmaceutical industry, MD

JR-171 is a blood brain-barrier (“BBB”)-penetrating form of recombinant α-L-iduronidase that was developed using JCR’s proprietary J-Brain Cargo® technology.

Key Points: 
  • JR-171 is a blood brain-barrier (“BBB”)-penetrating form of recombinant α-L-iduronidase that was developed using JCR’s proprietary J-Brain Cargo® technology.
  • There are no approved therapies that cross the BBB and address the central nervous system (CNS) symptoms for individuals with MPS I.
  • The overall safety data concluded that JR-171 is suitable for the long-term treatment of individuals with MPS I.
  • There is no impact on the consolidated business results for this fiscal year ending on March 31, 2024, related to the matter.

Orsini Specialty Pharmacy Partners with Amicus Therapeutics to Dispense New Therapy for the Treatment of Late-Onset Pompe Disease

Retrieved on: 
Thursday, September 28, 2023
Pharmacy, Glycogen, Glycogen storage disease, Miglustat, Amicus, Glycogen storage disease type II, Cell, Skeletal muscle, Muscle weakness, ELK, GAA, ERT, Infant, Patient, Cipaglucosidase alfa, Amicus Therapeutics, Enzyme replacement therapy, Disease, Pharmaceutical industry

ELK GROVE VILLAGE, Ill., Sept. 28, 2023 /PRNewswire/ -- Orsini Specialty Pharmacy has been selected by Amicus Therapeutics to dispense Pombiliti™ (cipaglucosidase alfa-atga) and Opfolda™ (miglustat), a two-component treatment approved for certain patients with late-onset Pompe disease.

Key Points: 
  • ELK GROVE VILLAGE, Ill., Sept. 28, 2023 /PRNewswire/ -- Orsini Specialty Pharmacy has been selected by Amicus Therapeutics to dispense Pombiliti™ (cipaglucosidase alfa-atga) and Opfolda™ (miglustat), a two-component treatment approved for certain patients with late-onset Pompe disease.
  • Please see full Prescribing Information, including BOXED WARNING, for POMBILITI and full Prescribing Information for OPFOLDA , also available at amicusrx.com .
  • Pompe disease is an inherited lysosomal disorder caused by a deficiency of the enzyme acid alpha-glucosidase.
  • We are pleased to partner with Amicus to bring Pombiliti and Opfolda to patients suffering from this devastating disease.

Muscular Dystrophy Association Celebrates FDA Approval of Amicus Therapeutics’ Pombiliti + Opfolda for Treatment of Pompe Disease

Retrieved on: 
Thursday, September 28, 2023
Duke University, US Food Sovereignty Alliance, University, Pediatrics, Doctor of Philosophy, Public policy, B cell, Glycogen storage disease type II, Medicine, Associate, Science, Capsule, ERT, Patient, Muscular Dystrophy Association, Blood, Amicus Therapeutics, MDA, FDA, Enzyme replacement therapy, Pharmaceutical industry, Cipaglucosidase alfa

New York, Sept. 28, 2023 (GLOBE NEWSWIRE) -- The Muscular Dystrophy Association (MDA) celebrates the US Food and Drug Administration (FDA) approval of Pombiliti™ (cipaglucosidase alfa-atga) + Opfolda™ (miglustat) 65mg capsules for adults living with late-onset Pompe disease (LOPD).

Key Points: 
  • New York, Sept. 28, 2023 (GLOBE NEWSWIRE) -- The Muscular Dystrophy Association (MDA) celebrates the US Food and Drug Administration (FDA) approval of Pombiliti™ (cipaglucosidase alfa-atga) + Opfolda™ (miglustat) 65mg capsules for adults living with late-onset Pompe disease (LOPD).
  • Pombiliti™ + Opfolda™ will be made available and marketed in the United States by Amicus Therapeutics.
  • Pombiliti™ + Opfolda™ is a novel two-component therapy consisting cipaglucosidase alfa-atga, a long-term enzyme replacement therapy (ERT), and miglustat, an oral enzyme stabilizer.
  • "We are extremely pleased with the recent approval of Pombiliti and Opfolda,” said Sharon Hesterlee, Ph.D., Chief Research Officer, MDA.

CompTIA Launches Emergency Response Team to Connect Cybersecurity Experts with Service Providers in Need of Assistance

Retrieved on: 
Wednesday, September 13, 2023
Workforce, Insurance, Technology specialist, Cioffi, Information technology, Intelligence, Cobham Technical Services, Organization, ERT, Incident response team, Health, CompTIA, Risk management, Management, PAX8

The new Emergency Response Team (ERT) is a group of dedicated CompTIA members who have either experienced a security incident or can provide guidance to solution providers that have been victimized.

Key Points: 
  • The new Emergency Response Team (ERT) is a group of dedicated CompTIA members who have either experienced a security incident or can provide guidance to solution providers that have been victimized.
  • Businesses in need of assistance can call (630) 678-8400 to be connected to a professional.
  • In addition to the new initiative, CompTIA continues to support the industry’s cybersecurity efforts with the CompTIA Information Sharing and Analysis Organization (ISAO) .
  • The CompTIA ISAO works closely with public and private cybersecurity agencies and organizations to help its members raise the cybersecurity awareness of the global tech industry.

Initial Clinical Data of First Pediatric CLN2 Patient Dosed with RGX-181 Presented at SSIEM Annual Symposium

Retrieved on: 
Wednesday, August 30, 2023
Seizure, Society, Therapy, TPP1, Neurodegenerative disease, Kia Challenge, Gene expression, Accelerated approval (FDA), Language, AAV, Hunter syndrome, Batten disease, Death, ERT, Patient, Child, Hospital, Enzyme replacement therapy, Safety, CLN2, BLA, Report of the 1st Annual Symposium on Relaxed Improvisation, Peptide, Disease, Pharmaceutical industry, Metabolism

"CLN2 is a debilitating disease caused by mutations in the CLN2 gene resulting in a deficiency of the TPP1 enzyme, which is needed to break down specific peptides associated with cellular waste.

Key Points: 
  • "CLN2 is a debilitating disease caused by mutations in the CLN2 gene resulting in a deficiency of the TPP1 enzyme, which is needed to break down specific peptides associated with cellular waste.
  • "We are encouraged by the initial results demonstrating that RGX-181 is well tolerated and dramatically reduced the number of seizures in the patient enrolled in this trial."
  • "The remarkable decrease in seizures, encouraging safety results and reduction in ERT frequency highlight the potential of this gene therapy to provide a meaningful treatment option to the CLN2 patient community."
  • Today, a physician investigator from the Hospital de Clinicas in Porto Alegre, Brazil reported initial results from a five-year-old child who received a one-time intracisternal dose of RGX-181.

Outcomes Data Reveal Significant Impact of Etiometry Utilization in Liberating Patients from Invasive ICU Treatments

Retrieved on: 
Monday, August 28, 2023
Managed Care, Software, Research, Technology, Hospitals, Health Technology, Surgery, Clinical Trials, Science, Cardiology, Nursing, Practice Management, Health, Hospice and palliative medicine, Risk, University of Alabama, Probability, University, HLA, Lactic acid, Perfusion, MSHA, Quality, Associate, Annals of Pediatric Cardiology, Harvard Medical School, Charles Mullins (pediatric cardiologist), Congress, Hospital, ERT, Metabolic acidosis, Assistant professor, Medical director, Patient, Division, Algorithm, Disease, ICU, Analytics, FDA, Weaning, MPH, Pelvic floor dysfunction, Automation, Medical device, Pharmaceutical industry, Pediatrics, MD

Etiometry’s Risk Analytics provide an individualized assessment of the patient’s condition, bringing attention to patients who need it most and revealing insight into the cause of potential deterioration or improvement to a patient's state.

Key Points: 
  • Etiometry’s Risk Analytics provide an individualized assessment of the patient’s condition, bringing attention to patients who need it most and revealing insight into the cause of potential deterioration or improvement to a patient's state.
  • Extubation failure is associated with significant morbidity, including prolonged total ventilation time and longer hospital length of stay.
  • The findings reveal the new automated standardized workflow in Etiometry informed data-driven decisions leading to an 18% reduction of time with vasoactive infusions.
  • The data are compelling, especially in terms of how our clinical intelligence can enhance patient care,” said Shane Cooke, CEO of Etiometry.

Fabry Disease Industry Insights 2023-2032: Epidemiology, Drug Pipeline, Emerging Treatments, Market Trends, Expert Opinions - ResearchAndMarkets.com

Retrieved on: 
Friday, August 25, 2023
Health, Other Health, EU4, Subject-matter expert, Chiesi Farmaceutici, Internet, USD, AAV, Protalix BioTherapeutics, ERT, GLA, Sangamo Therapeutics, Growth, Late onset congenital adrenal hyperplasia, Classic, Enzyme replacement therapy, Epidemiology, PEGylation, SME, Pharmaceutical industry, Vaccine, Video game, Dentistry, Fabry, EU, Fabry disease

This comprehensive analysis offers valuable insights into the Fabry Disease market, including epidemiology, drug pipeline, emerging treatments, market trends, and expert opinions, ultimately assisting stakeholders in understanding the evolving landscape and potential opportunities.

Key Points: 
  • This comprehensive analysis offers valuable insights into the Fabry Disease market, including epidemiology, drug pipeline, emerging treatments, market trends, and expert opinions, ultimately assisting stakeholders in understanding the evolving landscape and potential opportunities.
  • The report provides a comprehensive understanding of Fabry Disease epidemiology, including historical and forecasted data.
  • The Fabry Disease market experienced 15,290 diagnosed prevalent cases in the 7MM in 2022, with 8,355 cases in the US alone.
  • The Fabry Disease market size is projected to increase at a CAGR of 6.3% from 2023 to 2032, driven by awareness and the launch of emerging drugs.