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Pharming Group NV: Pharming announces publication of data from Phase 3 Study of leniolisib in patients with APDS in ASH's Blood

Leniolisib was well tolerated and significant improvement over placebo was notable in the co-primary endpoints, reflecting a favorable impact on patients' immune dysregulation and deficiency The ....

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Pharming Announces New ICD-10-CM Code for APDS, a Rare Primary Immunodeficiency

/PRNewswire/ Pharming Group N.V. ("Pharming" or "the Company") (EURONEXT Amsterdam: PHARM/Nasdaq: PHAR) announces that a new diagnosis code for reporting. ....

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Chiesi Global Rare Diseases Announces Support for Jeffrey Modell Foundation's Global Genetic Sequencing Initiative "Jeffrey's Insights"

Press release content from PR Newswire. The AP news staff was not involved in its creation. ....

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UCLA researchers develop base editing approach for rare genetic immune disorder

New research indicates the novel gene editing technique base editing could be used as a one-time therapy to restore the production of T cells in patients born with the rare genetic immune disorder CD3 delta severe combined immunodeficiency. This condition, also known as CD3 delta SCID, is caused by a mutation in the CD3 delta gene, which produces a protein by the same name ....

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Study sheds light on why immunodeficiency affects only one identical twin

Scientists have long queried the causes of immune disorders in only one of two identical twins with identical genes. ....

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