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Follow on Google News | Rare Disease Research: from Diagnostic Development to Small Molecule Drug DevelopmentBy: Protheragen One of the biggest hurdles in treating rare diseases is accurately diagnosing them. Many of these conditions have nonspecific symptoms that overlap with more common illnesses, leading to misdiagnosis and delayed treatment. Therefore, diagnostic development for rare diseases (https://www.protheragen.us/ Following with the early detection and diagnosis of rare diseases, the next step is developing effective treatments. Excitingly, recent advancements in gene engineering and drug R&D have led to the development of rare disease gene therapy and small-molecule drugs. Commonly, gene engineering technologies used in gene therapy include CRISPR/Cas9, zinc finger nucleases (ZFNs), transcription activator-like effector nucleases (TALEN), and meganucleases. These technologies offer hope to accelerate the research and development of rare disease therapies. Developing rare disease drugs is a complex and challenging process that often requires collaboration between researchers, pharmaceutical companies, and regulatory agencies. Usually, the drug development process of small molecule drugs includes identification and validation of initial targets, screening of lead compounds, method development and modeling, pharmacokinetic characterization, preclinical safety assessment, selection of preclinical drug candidates, and clinical studies. However, due to the small number of rare disease patients, traditional drug development approaches may not be economically viable. To address this issue, researchers have turned to innovative strategies, such as repurposing existing drugs, using artificial intelligence to screen potential compounds, and partnering with CRO companies working on rare disease research (https://www.protheragen.us/ End
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