Immunological Factors in Pompe Disease Management: Clinical Experience and Implications for Newborn Screening.

J Neuromuscul Dis

Division of Therapeutic Proteins, Office of Biotechnology Products, Center for Drug Evaluation and Research, United States Food and Drug Administration.

Published: January 2015

Download full-text PDF

Source

Publication Analysis

Top Keywords

immunological factors
4
factors pompe
4
pompe disease
4
disease management
4
management clinical
4
clinical experience
4
experience implications
4
implications newborn
4
newborn screening
4
immunological
1

Similar Publications

Perioperative Chemotherapy or Preoperative Chemoradiotherapy in Esophageal Cancer.

N Engl J Med

January 2025

From Bielefeld University, Medical School and University Medical Center Ostwestfalen-Lippe, Campus Hospital Lippe, Detmold, Germany (J.H.); the Department of Radiation Oncology, Medical University of Graz, Graz, Austria (T.B.); the Clinical Trials Unit, Faculty of Medicine and Medical Center, University of Freiburg, Freiburg, Germany (C.S.); the Institute of Surgical Pathology, University Medical Center Freiburg, Germany (P.B.); the Department of Surgery, University Medical Center Schleswig-Holstein-Campus Lübeck, Lübeck, Germany (B.K., T.K.); Comprehensive Cancer Center Augsburg, Faculty of Medicine, University of Augsburg, Augsburg, Germany (R.C.); the Department of General and Visceral Surgery, University Medical Center Freiburg, Freiburg, Germany (S.U.); the Department of General, Visceral, and Thoracic Surgery, University Medical Center Hamburg-Eppendorf, Hamburg, Germany (J.R.I.); the Department of Gastrointestinal Surgery, IRCCS San Raffaele Scientific Institute and San Raffaele Vita-Salute University, Milan (I.G.); the Department of General, Visceral, Thoracic, and Endocrine Surgery, Johannes Wesling University Hospital Minden, Ruhr University Bochum, Minden, Germany (B.G.); the Department of General, Visceral, and Pediatric Surgery, University Medical Center Göttingen, Göttingen, Germany (M.G.); the Department of General, Visceral, Thoracic, Transplantation, and Pediatric Surgery, University Medical Center Schleswig-Holstein-Campus Kiel, Kiel, Germany (B.R.); the Department of General, Visceral, Transplantation, Vascular, and Pediatric Surgery, University Hospital, University of Würzburg, Würzburg, Germany (J.F.L.); the Department of General, Visceral, Cancer, and Transplantation Surgery, University Hospital of Cologne, Cologne, Germany (C.B.); the Department of Hematology and Oncology, Sana Klinikum Offenbach, Offenbach am Main, Germany (E.R.); the Department of Surgery, Klinikum Dortmund, Klinikum der Universität Witten-Herdecke, Dortmund, Germany (M.S.); the Department of Surgery, University Hospital Magdeburg, Magdeburg, Germany (F.B.); the Department of Medicine I, University Hospital Carl Gustav Carus, Technical University Dresden, Dresden, Germany (G.F.); the Department of Hematology, Oncology, and Cancer Immunology, Charité-University Medicine Berlin, Campus Virchow-Klinikum, Berlin (P.T.-P.); the Department of General, Visceral, Cancer, and Transplantation Surgery, University Hospital Essen, Essen, Germany (U.P.N.); the Department of General, Visceral, and Transplantation Surgery, University Hospital Muenster, Muenster, Germany (A.P.); the Department of Radiotherapy and Oncology, Goethe University Frankfurt, University Hospital, Frankfurt, Germany (D.I.); the Division of Gastroenterology, Rheumatology, and Infectology, Department of Medicine, Charité-Universitätsmedizin Berlin, Berlin (S.D.); the Department of Surgery, Robert Bosch Hospital, Stuttgart, Germany (T.S.); the Department of Surgery, University Medical Center Erlangen, Friedrich Alexander University Erlangen-Nürnberg, Erlangen, Germany (C.K.); the Department of Medicine II, Saarland University Medical Center, Saarland University, Homburg, Germany (S.Z.); the Department of General, Visceral, and Transplant Surgery, Ludwig Maximilian University Hospital, Munich, Germany (J.W.); the Department of Internal Medicine I, Klinikum Mutterhaus der Borromaerinnen, Trier, Germany (R.M.); the Departments of Hematology, Oncology, and Palliative Care, Klinikum Stuttgart, Stuttgart, Germany (G.I.); the Department of General, Visceral, and Transplant Surgery, University Medical Center Mainz, Mainz, Germany (P.G.); and the Department of Medicine II, University Cancer Center Leipzig, Cancer Center Central Germany, University Medical Center Leipzig, Leipzig, Germany (F.L.).

Background: The best multimodal approach for resectable locally advanced esophageal adenocarcinoma is unclear. An important question is whether perioperative chemotherapy is preferable to preoperative chemoradiotherapy.

Methods: In this phase 3, multicenter, randomized trial, we assigned in a 1:1 ratio patients with resectable esophageal adenocarcinoma to receive perioperative chemotherapy with FLOT (fluorouracil, leucovorin, oxaliplatin, and docetaxel) plus surgery or preoperative chemoradiotherapy (radiotherapy at a dose of 41.

View Article and Find Full Text PDF

Association of poultry vaccination with interspecies transmission and molecular evolution of H5 subtype avian influenza virus.

Sci Adv

January 2025

State Key Laboratory of Remote Sensing Science, National Key Laboratory of Intelligent Tracking and Forecasting for Infectious Diseases, Beijing Research Center for Respiratory Infectious Diseases, School of National Safety and Emergency Management, Center for Global Change and Public Health, Beijing Normal University, Beijing, China.

The effectiveness of poultry vaccination in preventing the transmission of highly pathogenic avian influenza viruses (AIVs) has been debated, and its impact on wild birds remains uncertain. Here, we reconstruct the movements of H5 subtype AIV lineages among vaccinated poultry, unvaccinated poultry, and wild birds, worldwide, from 1996 to 2023. We find that there is a time lag in viral transmission among different host populations and that movements from wild birds to unvaccinated poultry were more frequent than those from wild birds to vaccinated poultry.

View Article and Find Full Text PDF

Background: Francisella tularensis is an aerobic, gram negative coccobacillus bacterium that causes tularemia. F. tularensis spreads primarily through ticks, biting flies, droplet inhalation, contaminated mud or water, or infected animal bites, and it can survive in animal carcasses with the most common mode of transmission occurring via inoculation into the skin and inhalation/ingestion.

View Article and Find Full Text PDF

Diphtheria antitoxin treatment: from pioneer to neglected.

Mem Inst Oswaldo Cruz

January 2025

Institut Pasteur, Université Paris Cité, Biodiversity and Epidemiology of Bacterial Pathogens, Paris, France.

Diphtheria, a severe respiratory infection, was a major killer of children until the early years of the 20th century. Although diphtheria is now largely controlled globally thanks to vaccination, it is still endemic in some world regions and large epidemics can occur where vaccination coverage is insufficient. The pathological effects caused by its main virulence factor, diphtheria toxin, can be diminished by passive transfer of antibodies.

View Article and Find Full Text PDF

One approach for developing a more universal influenza vaccine is to elicit strong immune responses against canonically immunosubdominant epitopes in the surface exposed viral glycoproteins. While standard vaccines typically induce responses directed primarily against mutable epitopes in the hemagglutinin (HA) head domain, there are generally limited or variable responses directed against epitopes in the relatively more conserved HA stalk domain and neuraminidase (NA) proteins. Here we describe a vaccine approach that utilizes a combination of wildtype (WT) influenza virus particles along with virus particles engineered to display a trimerized HA stalk in place of the full-length HA protein to elicit both responses simultaneously.

View Article and Find Full Text PDF

Want AI Summaries of new PubMed Abstracts delivered to your In-box?

Enter search terms and have AI summaries delivered each week - change queries or unsubscribe any time!