1st 2 weeks: gIFN 100 ugr/three times a week From the 3rd week: gIFN 200 ugr three times a week for the following 22 weeks From the 25th week: no treatment for the following 24 weeks

Friedreich Ataxia

1st two weeks: gIFN 100 ugr/three times a week from the 3rd week: gIFN 200 ugr/ three times a week for the following 22 weeks From the 25th week: no treatment for the following 24 weeks

gamma interferon

Open Trail of γIFN for Friedreich Ataxia

Safety and Efficacy of γIFN Treatment in Friedreich Ataxia

number of AE and SAE reported by treated patients along the study. Safety of γIFN treatment given for 6 months at final dose of 200 mcg three times weekly in FRDA patients

changes in scores at the SARA scale assessed at various time points (from 6 moths before treatment start to 6 months after treatment stop) for each patient

intensity of BOLD signal in FRDA patients performing the finger tapping protocol at least 6 months prior to treatment start, at treatmenet start, after 6 months of treatment and 6 months after treatment discontinuation

Mean Thickness of RNFL and thickness in the 4 quadrants as resulting from OCT exam

changes in ventricular and interventricular walls thickness (mm) at various time points for each patient

changes in Frataxin levels (intensity of frataxin band at western blot) associated with treatment

scores at SF36 assessed along time in each patient

Fractional anysotropy values at selected ROI as measured at various time points in each patient

changes in WHO-DAS 2.0 scores recorede along time

MD values at selected ROI as measured at various time points in each patient

Andrea Martinuzzi

Director of neurorehabilitation department

IRCCS Eugenio Medea

Open label = Single dose administration of single dose Denosumab 120 mg subcutaneously

Crohn Disease

Denosumab

University of Manitoba

The Efficacy of Denosumab in Active Crohn's Disease

Denosumab (A Monoclonal Antibody to Receptor Activator of Nuclear Factor-Kappa B Ligand (RANKL) in Crohn's Disease

Decrease in CDAI to ≤150 points at 12 weeks.

In those with elevated fecal calprotectin at baseline, reduction in fecal calprotectin to <250 ug/g at week 12.

for those with increased CRP at baseline change in CRP to normal at week 12

For those who underwent endoscopy within 1 month of study enrollment as part of standard of care and who underwent a repeat endoscopy within 1 month of study completion as part of standard of care, improvement in endoscopy by Crohn's Disease Endoscopy Inflammation Score (CDEIS) between baseline and second endoscopy.

For those who underwent abdominal MRI within 1 month of study enrollment as part of standard of care and who underwent a repeat abdominal MRI within 1 month of study completion as part of standard of care then improvement in MRI findings by central reader (Dr H Greenberg) between baseline and second abdominal MRI.

Serum calcium will be assessed at 3 days post drug administration and every 4 weeks. There is a rare incidence of hypocalcemia with use of this drug. Osteonecrosis of the jaw will be assessed at every study visit. This drug is rarely associated with this outcome especially in cancer patients. Liver enzymes every 4 weeks will be assessed.

osteopetrosis/ألبيومين

Open Trail of γIFN for Friedreich Ataxia

The Efficacy of Denosumab in Active Crohn's Disease

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