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Glaxo will also work with the Italian researchers to develop the gene therapy for six other rare diseases.
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Patients' families with other rare diseases also are seeking to press researchers to share data from natural-history studies.
WSJ: Families Push for New Ways to Research Rare Diseases
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Moreover, repositioning such drugs to treat other rare diseases can ring more registers.
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Since then, Tolar has transplanted a total of 24 children with EB, and continues to conduct research in gene therapy for EB and other rare diseases.
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But a lot of credit for this recent bounce should go to the FDA itself, which under Commissioner Margaret Hamburg has worked more closely with companies to streamline the approval process, particularly in cancer and other rare diseases.
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These drugs to treat rare cancers and other uncommon diseases were approved very quickly by the FDA, then went on to reap big sales.
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Shire is a pharmaceutical company that develops health care in the areas of behavior health and gastrointestinal conditions, rare diseases and regenerative medicine and other conditions.
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He has also brought clarity and insight to a number of other curious brain diseases, including the very rare syndrome where a patient believes his or her mother to be an imposter.
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First, drugs are increasingly a specialty business, with companies focusing on treatments for genetic diseases, uncommon cancers, and other rare ailments.
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Such super-high-priced drugs for ultra-rare diseases are the biggest success story in pharma right now (see BioMarin and even Genzyme for other examples).
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