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Discarded Bone Marrow for Hematology Research
The primary objective of this study is to establish a mechanism to obtain discarded bone marrow-containing bone samples from hemoglobinopathy, as well as non-h…
Effectiveness and Safety of Sotagliflozin in Slowing Kidney Function Decline in Persons With Type 1 Diabetes and Moderate to Severe Diabetic Kidney Disease
Powerful new drugs that can prevent or delay end stage kidney disease (ESKD) - so called sodium-glucose cotransporter-2 inhibitors (SGLT2i) - are now available…
The Development of Minimally Invasive Nanosensor Technology to Quantify Mitochondrial Function in Human Muscle
Past mitochondrial disease treatment studies have been unsuccessful in determining treatment efficacy, and a major factor has been the lack of validated biomar…
Tailored Exercise Training Study Among Adults With HFpEF
Heart failure with preserved ejection fraction (HFpEF) is associated with a high morbidity and mortality burden. There are limited pharmacological options avai…
Optimizing Beta Blocker Dosage in Women While Using the Wearable Cardioverter Defibrillator
The primary objective of this pilot study is to document the percentage achievement in effective HR control (average nighttime HR \< 70 bpm) during WCD use in …
A Patient Registry and Natural History Study of Patients with Biallelic HPDL Mutations
This study uses medical records that allow retrospective data extraction of clinical manifestation to assess the natural history of HPDL mutations