One of the less well known forms of primary immunodeficiency is called activated PI3K delta syndrome (APDS). It is a rare disease that has only recently been identified by scientists.…
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The gene therapy Zolgensma recently won approval last summer in 2019 to be used for infants who have spinal muscular atrophy. The company AveXis, the makers of the Zolgensma gene…
CSL Behring has recently received the Orphan Drug designation for Privigen for the treatment of systemic scleroderma. Privigen has also been granted the Fast Track designation. This therapy has been…
Teva Pharmaceuticals recently finished two clinical trials of their Tourette syndrome treatment, deutetrabenazine. These two studies, titled ARTISTS 1 and ARTISTS 2, did not meet their primary endpoints, as they…
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