Working together to improve the outcome for patients with rare, life-threatening, inherited disorders of the immune system.

 

 

Research Studies

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See opportunities to participate in research and help further innovations in care for patients like you.

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Learn About Newborn Screening

Infants with SCID who are transplanted before 3 months of age fare better than infants who are transplanted later in life. Learn more about newborn screenings for SCID and help improve outcomes for this rare disorder in the future.

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Rare Research Report: January 2023

Each month, we share summaries of recent Rare Diseases Clinical Research Network (RDCRN) grant-funded publications. Catch up on the latest RDCRN research. 

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PIDTC/IEWP STAT1 GOF HSCT STUDY - Now open!

On behalf of the PIDTC 6906 Project Working Team and in collaboration with the IEWP, we are collecting data to assess the outcomes of patients with STAT1 Gain of Function of any age who have undergone hematopoietic cell transplantation since 2010, including survivors and non-survivors with any length of follow-up.

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Artemis-SCID Gene Therapy Featured in UCSF Video

Artemis-SCID is a severe form of immunodeficiency caused by mutations in a specific gene.

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Jennifer Puck, MD

PIDTC Updates

Newsletter of the Primary Immune Deficiency Treatment Consortium

Issue 17 | Winter

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