174 results on '"Cattaneo, Federica"'
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2. Comprehensive long‐term efficacy and safety of recombinant human alpha‐mannosidase (velmanase alfa) treatment in patients with alpha‐mannosidosis
3. The actigraphic documentation of circadian sleep-wake rhythm dysregulation in myotonic dystrophy type 1
4. Use of the Bruininks-Oseretsky test of motor proficiency (BOT-2) to assess efficacy of velmanase alfa as enzyme therapy for alpha-mannosidosis
5. Efficacy of a Fosfomycin-Containing Regimen for Treatment of Severe Pneumonia Caused by Multidrug-Resistant Acinetobacter baumannii: A Prospective, Observational Study
6. Intraoperative identification of the laryngeal corticomotor region.
7. Impaired Visual Inhibition in Amnestic Mild Cognitive Impairment.
8. Enzyme replacement therapy with velmanase alfa (human recombinant alpha-mannosidase): Novel global treatment response model and outcomes in patients with alpha-mannosidosis
9. Gene therapy with adeno-associated virus vector 5–human factor IX in adults with hemophilia B
10. Correction of ADA-SCID by Stem Cell Gene Therapy Combined with Nonmyeloablative Conditioning
11. The SPARKLE registry: protocol for an international prospective cohort study in patients with alpha-mannosidosis
12. WASP regulates suppressor activity of human and murine CD4+CD25+FOXP3+ natural regulatory T cells
13. Efficacy and safety of Velmanase alfa in the treatment of patients with alpha-mannosidosis: results from the core and extension phase analysis of a phase III multicentre, double-blind, randomised, placebo-controlled trial
14. Impaired Visual Inhibition in Amnestic Mild Cognitive Impairment
15. Hypomorphic Janus kinase 3 mutations result in a spectrum of immune defects, including partial maternal T-cell engraftment
16. IL-21 is the primary common γ chain-binding cytokine required for human B-cell differentiation in vivo
17. Revertant T lymphocytes in a patient with Wiskott-Aldrich syndrome: Analysis of function and distribution in lymphoid organs
18. Impaired visual inhibition in amnestic mild cognitive impairment
19. Assessment of self-reported and objective daytime sleepiness in adult-onset myotonic dystrophy type 1
20. Altered intracellular and extracellular signaling leads to impaired T-cell functions in ADA-SCID patients
21. Efficacy of Daptomycin-Containing Regimen for Treatment of Staphylococcal or Enterococcal Vertebral Osteomyelitis: A Prospective Clinical Experience
22. Comparison Between Hospitalized Patients Affected or Not Affected by Coronavirus Disease 2019
23. Efficacy of a Fosfomycin-Containing Regimen for Treatment of Severe Pneumonia Caused by Multidrug-Resistant Acinetobacter baumannii: A Prospective, Observational Study
24. The SPARKLE registry: protocol for an international prospective cohort study in patients with alpha-mannosidosis
25. Clinical features and outcome of difficult-to-treat infections in a high-intensity medical care ward
26. Velmanase alfa enzyme replacement therapy for alpha-mannosidosis improves patient outcomes over standard of care both in terms of clinically relevant improvement and disease stabilization
27. A new randomized placebo-controlled study to establish the safety and efficacy of velmanase alfa (human recombinant alpha-mannosidase) enzyme replacement therapy for the treatment of alpha-mannosidosis
28. Clinical features and outcome of difficult-to-treat infections in a high-intensity medical care ward.
29. Gene Therapy for Immunodeficiency Due to Adenosine Deaminase Deficiency
30. Comparison between hospitalized patients affected or not by COVID-19 (RESILIENCY study)
31. Clinical improvement and normalized Th1 cytokine profile in early and long-term interferon-α treatment in a suspected case of hyper-IgE syndrome
32. The SPARKLE registry: a multicenter, multinational, noninterventional, prospective cohort registry study in patients with alpha-mannosidosis
33. The SPARKLE study: Shedding light on alpha mannosidosis
34. The first study investigating safety and efficacy of velmanase alfa (human recombinant alpha mannosidase) in alpha-mannosidosis patients below six years of age
35. DISTURBI DEL SONNO E SALUTE MENTALE NEI LAVORATORI OSPEDALIERI DURANTE LA PANDEMIA DI COVID-19: UNO STUDIO MULTICENTRICO TRASVERSALE NEL NORD ITALIA.
36. Gender, mutations and residual enzymatic activity: Investigation of predictive factors of alpha-mannosidosis phenotypic presentation and of response to velmanase alfa long term enzyme replacement therapy
37. Global treatment responder analysis demonstrates clinically relevant effect of velmanase alfa long term enzyme replacement therapy for alpha mannosidosis, in a phase III randomized placebo controlled trial
38. Global treatment response analysis of velmanase alfa long term enzyme replacement therapy for alpha-mannosidosis shows treatment benefit across ages
39. Health Related Quality of Life, Disability, and Pain in Alpha Mannosidosis
40. Improvement in pulmonary function and serum immunoglobulin G in long-term enzyme replacement therapy with velmanase alfa(human recombinant alpha-mannosidase) in alpha-mannosidosis patients
41. Long-term enzyme replacement therapy with velmanase alfa (human recombinant alpha-mannosidase) improves mobility in alpha-mannosidosis patients
42. Long-term enzyme replacement therapy with velmanase alfa (human recombinant alpha-mannosidase) slows disease progression in adult patients suffering from alpha-mannosidosis
43. Improvement in fine and gross motor proficiency after long-term enzyme replacement therapy with velmanase alfa (human recombinant alpha mannosidase) in alpha-mannosidosis patients
44. Interim Results from a Dose Escalating Study of AMT-060 (AAV5-hFIX) Gene Transfer in Adult Patients with Severe Hemophilia B
45. Abstract LB-197: First-in-human study with ARQ 092, a novel pan AKT-inhibitor: Results from the advanced solid tumors cohorts.
46. Somatic Gene Therapy for X-Linked Severe Combined Immunodeficiency Using a Self-Inactivating Modified Gammaretroviral Vector Results in An Improved Preclinical Safety Profile and Early Clinical Efficacy in a Human Patient
47. Optimal Thalassemia Free Survival and Minimal Regimen Related Toxicity in 50 Consecutive Transplants of High Risk Beta Thalassemia Pediatric Patients Using Myelablative Therapy with Intravenous Busulphan.
48. Long-Term Safety and Efficacy of Stem Cell Gene Therapy for ADA-SCID.
49. Deoxyadenosine Inhibits T-Cell Activation in ADA-SCID Patients through a cAMP/PKA-Dependent Pathway.
50. 672. Lentiviral Vectors Targeting WASp Expression to Hematopoietic Cells, Efficiently Transduce CD34+ Cells and Correct Functions of Lymphocytes and Dendritic Cells from Wiskott- Aldrich Syndrome Patients
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