Results 11 to 20 of about 117,553 (304)
Expression of γ-globin genes in β-thalassemia patients treated with sirolimus: results from a pilot clinical trial (Sirthalaclin) [PDF]
Introduction: β-thalassemia is caused by autosomal mutations in the β-globin gene, which induce the absence or low-level synthesis of β-globin in erythroid cells.
Cristina Zuccato +17 more
openalex +2 more sources
BACKGROUND Slow-flow vascular malformations frequently harbor activating mutations in the PI3K/AKT/mTOR cascade. Phase II trials pinpointed sirolimus effectiveness as a drug therapy.
Emmanuel Seront +16 more
semanticscholar +1 more source
BACKGROUND Fibrocytes are BM-derived circulating cells that traffic to the injured lungs and contribute to fibrogenesis. The mTOR inhibitor, sirolimus, inhibits fibrocyte CXCR4 expression, reducing fibrocyte traffic and attenuating lung fibrosis in ...
Diana C. Gomez Manjarres +6 more
semanticscholar +1 more source
The Kasabach-Merritt phenomenon (KMP) in kaposiform hemangioendothelioma (KHE) is characterized by life-threatening thrombocytopenia and consumptive coagulopathy. This study compared the efficacy and safety of sirolimus plus prednisolone versus sirolimus
Yi Ji +13 more
semanticscholar +1 more source
nab-Sirolimus for Patients With Malignant Perivascular Epithelioid Cell Tumors
PURPOSE Malignant perivascular epithelioid cell tumor (PEComa) is a rare aggressive sarcoma, with no approved treatment. To our knowledge, this phase II, single-arm, registration trial is the first prospective clinical trial in this disease ...
A. Wagner +16 more
semanticscholar +1 more source
Efficacy of sirolimus in children with lymphatic malformations of the head and neck
Children with extensive lymphatic malformations of the head and neck often suffer from functional impairment and aesthetic deformity which significantly affect the quality of life and may be life-threatening. Treatment with sirolimus has the potential to
S. Wiegand, A. Dietz, G. Wichmann
semanticscholar +1 more source
Importance Sirolimus is increasingly being used to treat various vascular anomalies, although evidence of its efficacy is lacking. Objective To assess the efficacy and safety of sirolimus for children with slow-flow vascular malformations to better ...
A. Maruani +24 more
semanticscholar +1 more source
Sirolimus for epileptic seizures associated with focal cortical dysplasia type II
To determine whether sirolimus, a mechanistic target of rapamycin (mTOR) inhibitor, reduces epileptic seizures associated with focal cortical dysplasia (FCD) type II.
Mitsuhiro Kato +11 more
semanticscholar +1 more source
Multiple system atrophy (MSA) is a fatal neurodegenerative disease characterized by the aggregation of α‐synuclein in glia and neurons. Sirolimus (rapamycin) is an mTOR inhibitor that promotes α‐synuclein autophagy and reduces its associated ...
J. Palma +14 more
semanticscholar +1 more source
Sirolimus as a second-line treatment for Graves’ orbitopathy
A beneficial effect of sirolimus in Graves’ orbitopathy (GO) was reported, suggesting a possible use in clinical practice. We conducted an observational, single-centre, no-profit, clinical study to investigate the efficacy of sirolimus as a second-line ...
G. Lanzolla +8 more
semanticscholar +1 more source

