This is a Phase 2, open-label study to A Phase 2, Open-Label Study to Evaluate the Safety and Tolerability of Progerinin for the Treatment of Bone Mineral Density (BMD) Loss in Subjects with Typical Werner Syndrome.
There will be up to 5 subjects that will receive treatment with Progerinin twice daily for approximately 1 year.
Subjects must be at least ≥ 18 years of age.
Subjects should have a confirmed diagnosis of typical Werner syndrome by genetic analysis.
Subjects should be osteopenic (T-score between -1.0 and -2.5) or have confirmed osteoporosis (Tscore ≤ -2.5) at screening/baseline. Subjects may continue to receive bone supplements (e.g., bisphosphonates, calcium supplements, Vitamin D supplements, etc.) as appropriate per standard of care throughout the study.
Male or non-pregnant, non-lactating female.
Subjects who received continuous or intermittent home oxygen therapy for 6 months before obtaining consent
Subjects who received at least 2 hospitalizations for pneumonia during the 12 months prior to obtaining consent
Subjects who have at least 10% net weight loss and have not recovered. This includes significant net weight loss over the last six months.
Subjects with significant dehydration as judged by the principal investigator.