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Danon disease involves a genetic defect (mutation) in a gene called LAMP2, which results in a change to the normal protein structure. While the function of the LAMP2 gene is not well understood, it is known that LAMP2 protein is primarily located in small structures within cells called lysosomes .
The DRKS is an open access, free of charge online register for clinical trials and is available both in English and German. DRKS is part of the WHO's ICTRP. The DRKS works with two partner registries in Germany, DeReG (German Registry for Somatic Gene-Transfer Trials) and Clinical Trial Registry of the University Medical Center Freiburg. [4]
As a result of pressure from HIV-infected men in the gay community, [citation needed] who demanded better access to clinical trials, the U.S. Congress passed the Health Omnibus Programs Extension Act of 1988 (Public Law 100-607) [2] which mandated the development of a database of AIDS Clinical Trials Information Services (ACTIS). [3]
At its core is the collection of Cochrane Reviews, a database of systematic reviews and meta-analyses that summarize and interpret the results of medical research. The Cochrane Library aims to make the results of well-conducted clinical trials readily available and is a key resource in evidence-based medicine .
Gene therapy is another treatment option which is available only for clinical trials. [34] X-linked SCID is a monogenic disorder, the IL2RG gene is mutated, so gene therapy will replace this mutated gene with a normal one. [39] This will result in a normal functioning gamma chain protein of the interleukin receptor. [35]
The International Clinical Trials Registry Platform (ICTRP) is a platform for the registration of clinical trials operated by the World Health Organization. [1]The ICTRP combines data from multiple cooperating clinical trials registries to generate a global view of clinical trials worldwide, with a search portal that allows access to the entire dataset.
Abdi Nageeye outlasted 2022 champion Evans Chebet to win the men's 2024 New York City Marathon. Sheila Chepkirui won the women's race.
It was developed in response to Archie Cochrane's call for up-to-date, systematic reviews of all relevant randomised controlled trials in the field of healthcare. [11] [12] [13] In 1998, the Cochrane Economics Methods Group (CEMG) was established to facilitate the basing of decisions on health economics, evidence-based medicine, and systematic ...