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Rare Disease Day 2017 With A Commitment To Provide New Insights Into Rare Diseases Product Applications In Usa Massachusetts
4 applications found
Lysosomal diseases are a group of rare, genetic metabolic diseases in which cellular waste products build up in the body. These disorders are generally progressive and there is no cure. Many lysosomal diseases have no FDA-approved ...
ByEli Lilly and Company based in Indianapolis, INDIANA (USA)
The endocrine and metabolic system includes endocrine glands and endocrine tissues, which regulate human function by secreting hormones. Endocrine diseases are rare and often very difficult to diagnose and treat. Among the 121 rare diseases included on China’s national list of rare diseases in 2018, more than 20 are endocrine/metabolic-related rare ...
ByRareStone Group based in Shanghai, CHINA
Rare neurological diseases include developmental defects of the nervous system, genetic and metabolic diseases, neuroimmune diseases, neurological tumors, special types of infectious diseases of the nervous system, neurodegenerative diseases, and neuromuscular diseases. At present, more than 7,000 rare diseases have been found in humans, about 60% of which are neurological ...
ByRareStone Group based in Shanghai, CHINA
Using our platform, we are developing mRNA medicines for diverse uses and delivering them to patients across the globe. We are currently focusing on 4 medical areas - infectious diseases, immuno-oncology, rare diseases and autoimmune ...
ByModerna, Inc. based in Cambridge, MASSACHUSETTS (USA)