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Clinical evidence of interventions assessed in Friedreich ataxia: a systematic review.
Ther Adv Rare Dis
November 2022
PTC Therapeutics, Zug, Switzerland.
Objectives: The rare inherited autosomal recessive disease Friedreich ataxia (FA) causes progressive neurodegenerative changes and disability in patients. A systematic literature review (SLR) was carried out to understand and summarize the published efficacy and safety of therapeutic interventions in this disease.
Methods: Database searches were carried out in MEDLINE, Embase, and Cochrane by two independent reviewers.
The only treatment currently available for patients with severe infantile osteopetrosis is hematopoietic cell transplantation (HCT). HCT-related toxicity and mortality risks typically preclude its use in non-infantile patients, and other therapies are needed for these patients who have significant disease-related morbidity. Interferon gamma-1b is currently approved by the U.
View Article and Find Full Text PDFThe application of HPLC/MS analysis with a multi-enzyme digest strategy to characterize different interferon product variants produced from Pichia pastoris.
Amino Acids
September 2019
Barnett Institute and Department of Chemistry and Chemical Biology, Northeastern University, 360 Huntington Avenue, Boston, MA, 02115, USA.
Interferons are signaling proteins that belong to the large class of cytokines and human interferons which are classified based on the type of receptor interactions: type I, II and III. IFNα2b belongs to the type I interferon class with a major therapeutic application for the treatment of hepatitis B and C infections. A recombinant form of IFNα2b expressed in E.
View Article and Find Full Text PDFRandomized, double-blind, placebo-controlled study of interferon- 1b in Friedreich Ataxia.
Ann Clin Transl Neurol
March 2019
Friedreich's Ataxia Research Alliance 533 W Uwchlan Ave Downingtown Pennsylvania 19335.
Objective: In vitro, in vivo, and open-label studies suggest that interferon gamma (IFN- 1b) may improve clinical features in Friedreich Ataxia through an increase in frataxin levels. The present study evaluates the efficacy and safety of IFN- 1b in the treatment of Friedreich Ataxia through a double-blind, multicenter, placebo-controlled trial.
Methods: Ninety-two subjects with FRDA between 10 and 25 years of age were enrolled.
Interferon Gamma-1b Does Not Increase Markers of Bone Resorption in Autosomal Dominant Osteopetrosis.
J Bone Miner Res
August 2019
Department of Medicine, Indiana University School of Medicine, Indianapolis, IN, USA.
In autosomal dominant osteopetrosis type 2 (ADO2) CLCN7 mutations cause impaired osteoclast function. Severe consequences include skeletal fragility despite high bone mass, osteomyelitis, osteonecrosis, bone marrow failure, and severe cranial nerve impingement. There is no effective medical treatment for ADO2.
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