Familial cardiomyopathy

This study tested an experimental drug called CRD-4730 in people with a rare inherited heart rhythm disorder (CPVT) that can cause dangerous fast heartbeats. Seven adults received two different doses of the drug and a placebo to check safety and how the body processes it. The stu…

This study tested a drug called CardiolRx in 90 hospitalized COVID-19 patients who also had heart disease or risk factors like diabetes or high blood pressure. The goal was to see if the drug could lower the chance of death, needing intensive care, or having heart problems. The t…

This early-stage trial tested an oral drug called TNO155, alone or with another drug (nazartinib), in 227 adults with advanced solid tumors like lung cancer, melanoma, and head/neck cancer. The main goal was to check safety and find the right dose. The study was terminated early,…

This study tested a gene therapy called SRP-9003 for people with limb-girdle muscular dystrophy type 2E (LGMD2E), a rare genetic disease that causes muscle weakness. The treatment aimed to deliver a working gene to muscle cells to help them produce a missing protein. Only 6 peopl…

This study tested a device called Efferon LPS that filters blood during heart surgery when a heart-lung machine is used. The goal was to see if it could prevent multiple organ failure, especially kidney damage. Only 14 people took part before the study was stopped, so the results…

This study tested an oral drug called duvoglustat in adults with Pompe disease, a rare genetic disorder that causes muscle weakness. Only 3 people took part, and the study was stopped early. Researchers looked at safety and whether the drug could improve walking distance and othe…

This early-stage trial tested a gene therapy called SRP-9004 in just 4 people with limb girdle muscular dystrophy type 2D/R3, a rare muscle-weakening disease. The main goal was to check safety, not effectiveness. The study was terminated early, so results are limited.

This study looked at whether letting patients manage their own recovery precautions after heart surgery leads to a better quality of life compared to following standard sternal precautions. Ten adults who had heart surgery were followed for up to a year. Researchers checked pain,…

This study collects small skin samples from children aged 2 to 17 with Costello syndrome or a related condition. Researchers will analyze the cells to understand how a mutation in the HRAS gene affects energy use and mitochondria. The goal is to learn more about the disease's und…

This study looked at how two common pain relievers—celecoxib (a Coxib) and naproxen (an NSAID)—affect blood clotting in people with rheumatoid arthritis who also have heart disease or are at high risk for it. All participants took low-dose aspirin daily. The goal was to see if on…

This study collected bone, cartilage, skin, and fat tissue from 50 patients undergoing surgery at Children's National Medical Center. The goal was to grow cells from these tissues in the lab and analyze them to better understand bone and connective tissue diseases. The study was …

This study aims to find out how common transthyretin amyloidosis cardiomyopathy (ATTR-CM) is in Russian patients with a certain type of heart failure. Researchers will review medical records and then invite some patients for extra heart tests to confirm or rule out ATTR-CM. The g…