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A rare disease is a disease that affects a small percentage of the population. In some parts of the world, the term orphan disease describes a rare disease whose rarity results in little or no funding or research for treatments, without financial incentives from governments or other agencies. Orphan drugs are medications targeting orphan ...
In 2012, to focus on newer, strategic CNS-products, Lundbeck sold a portfolio of non-core products to Recordati S.p.A. (Recordati Rare Diseases). [5] In 2014 Lundbeck acquired Chelsea Therapeutics for up to $658 million. [6] In March 2018, the company acquired Prexton Therapeutics for up to €905 million ($1.1 billion) [7]
2018 It was announced by Recordati Rare Diseases Inc. that due to a combination of low product demand and complex manufacturing difficulties, product manufacturing, distribution and sale was being discontinued.
Hypophosphatasia (/ ˌ h aɪ p oʊ ˈ f ɒ s f eɪ t ˌ eɪ ʒ ə /; also called deficiency of alkaline phosphatase, phosphoethanolaminuria, [5] or Rathbun's syndrome; [1] sometimes abbreviated HPP [6]) is a rare, and sometimes fatal, inherited [7] metabolic bone disease. [8]
ICD coding for rare diseases. The ICD coding for rare diseases is the International Classification of Diseases code used for the purpose of documenting rare diseases. It is important for health insurance reimbursement, administration, epidemiology, and research. Of the approximately 7,000 rare diseases, only about 500 have a specific code.
The Rare Diseases Clinical Research Network (RDCRN) [ 7] is a U.S.-based research network funded by the NIH. It fosters research to better understand, diagnose, and treat rare diseases. Its 20 consortia—teams of scientists, physicians, and patients—each study a group of related rare diseases. Established by Congress under the Rare Diseases ...
The following is a list of genetic disorders and if known, type of mutation and for the chromosome involved. Although the parlance "disease-causing gene" is common, it is the occurrence of an abnormality in the parents that causes the impairment to develop within the child. There are over 6,000 known genetic disorders in humans.
The Rare Diseases Clinical Research Network ( RDCRN) is funded by the National Institutes of Health (NIH) and led by the National Center for Advancing Translational Sciences (NCATS) through its Division of Rare Diseases Research Innovation (DRDRI). The RDCRN is designed to advance medical research on rare diseases by providing support for ...