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114 results
  • Open-Label Pilot Study of Interferon Gamma-1b in Patients With Non-Infantile Osteopetrosis. [Journal Article]
    JBMR Plus. 2022 Mar; 6(3):e10597.Nguyen A, Miller WP, … Polgreen LE
  • 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.S. Food and Drug Admin…
  • Designing phase II clinical trials in Friedreich ataxia. [Review]
    Expert Opin Emerg Drugs. 2021 12; 26(4):415-423.Rodden LN, Lynch DR
  • Friedreich ataxia (FRDA) is an autosomal recessive disorder caused by deficiency of frataxin, an essential mitochondrial protein involved in iron sulfur cluster biogenesis, oxidative phosphorylation and other processes. FRDA most notably affects the heart, sensory neurons, spinal cord, cerebellum, and other brain regions, and manifests clinically as ataxia, sensory loss, dysarthria, spasticity, a…
  • Drugs and Lactation Database (LactMed): Interferon Gamma-1b [BOOK]
    Drugs and Lactation Database (LactMed). National Library of Medicine (US): Bethesda (MD)BOOK
  • Interferon gamma is a normal component of human milk. No data are available on the use of exogenous interferon gamma 1b during breastfeeding. However, the amounts of the similar drugs, interferon alfa and interferon beta-1a, excreted into milk are very low. Any interferon in breastmilk is probably destroyed in the infant's gastrointestinal tract and not absorbed, except perhaps in neonates. Holde…
  • A rare complication of chronic granulomatous disease in a child: constrictive aspergillus pericarditis. [Case Reports]
    Turk Kardiyol Dern Ars. 2017 Oct; 45(7):660-663.Şişli E, Özdemir Şahan Y, … Atay Y
  • A 3-year-old girl with the diagnosis of chronic granulomatous disease (CGD) was hospitalized for bronchopneumonia and congestive heart failure. Her medical history included methylprednisolone medication for autoimmune gastric outlet obstruction. Computed tomography revealed pneumonic infiltrations and pericardial thickening. A pulsed-wave Doppler recording revealed E/A >1. During a pericardiectom…
  • IFN-γ for Friedreich ataxia: present evidence. [Journal Article]
    Neurodegener Dis Manag. 2015 Dec; 5(6):497-504.Wells M, Seyer L, … Lynch DR
  • IFN-γ-1b is currently US FDA approved as an orphan drug for the treatment of chronic granulomatous disease and severe malignant osteopetrosis. It is administered via subcutaneous injection and is a potential therapy for Friedreich ataxia (FRDA), a rare degenerative neurological condition. Ongoing Phase II and III trials in both adults and children with FRDA were preceded by a small Phase I, open-…
  • Pulmonary Fibrosis Treated with Inhaled Interferon-gamma (IFN-γ). [Case Reports]
    J Aerosol Med Pulm Drug Deliv. 2015 Oct; 28(5):406-10.Fusiak T, Smaldone GC, Condos R
  • Parenteral IFN-γ was unsuccessful as a treatment for pulmonary fibrosis. Inhaled IFN-γ targeted to the lungs may be more effective. Our patient, a 56-year-old male with biopsy proven usual interstitial pneumonia (UIP) and declining pulmonary function tests (PFTs) was initially diagnosed with idiopathic pulmonary fibrosis (IPF). He enrolled in a 2-year research protocol and was treated with inhale…
  • Inhaled Interferon and Diffusion Capacity in Idiopathic Pulmonary Fibrosis (IPF). [Randomized Controlled Trial]
    Sarcoidosis Vasc Diffuse Lung Dis. 2015 Jun 22; 32(1):37-42.Skaria SD, Yang J, … Smaldone GC
  • CONCLUSIONS: DLCO was significantly improved following inhaled (IFN-γ) as treatment for IPF. Our data suggest that previous studies utilizing parenteral IFN-γ may have failed because of the mode of delivery. Future randomized, controlled, phase 3 trials, comparing the difference in PFT behavior (specifically DLCO) longitudinally may be more sensitive to drug effect and serve as a valuable clinical endpoint.
  • Interferon Gamma, but not Calcitriol Improves the Osteopetrotic Phenotypes in ADO2 Mice. [Journal Article]
    J Bone Miner Res. 2015 Nov; 30(11):2005-13.Alam I, Gray AK, … Econs MJ
  • ADO2 is a heritable osteosclerotic disorder that usually results from heterozygous missense dominant negative mutations in the chloride channel 7 gene (CLCN7). ADO2 is characterized by a wide range of features and severity, including multiple fractures, impaired vision due to secondary bony overgrowth and/or the lack of the optical canal enlargement with growth, and osteonecrosis/osteomyelitis. T…
  • Open-label pilot study of interferon gamma-1b in Friedreich ataxia. [Clinical Trial]
    Acta Neurol Scand. 2015 Jul; 132(1):7-15.Seyer L, Greeley N, … Lynch DR
  • CONCLUSIONS: Interferon gamma-1b improved FARS scores without a clear relationship to changes in frataxin levels. Larger, longer placebo-controlled trials including biochemical assessments in affected tissues are necessary to evaluate fully the efficacy and utility of IFN-γ in FRDA.
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