Project ECHO clinician series: What have we learned?
July 24, 2023
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NIH, IDF support young adult in his journey with PI
July 19, 2023
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Volunteer as a community liaison
Your time and talents can transform the lives of others.
The Assistance Fund
The Assistance Fund is an independent 501(c)(3) organization that helps patients and families facing high medical out-of-pocket costs by providing financial assistance for their copayments, coinsurance, deductibles, and other health-related expenses.
Study shows newborn screening improved SCID survival rates
July 13, 2023
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Good Days
Good Days is a national non-profit charitable organization that lifts the burdens of chronic illness through assistance, advocacy, and awareness. Their financial assistance program for people with chronic granulomatous disease (CGD) providers up to $3500 for medication costs.
Waskyra
Etuvetidigene autotemcel.
Approved to treat: Wiskott-Aldrich syndrome (WAS) in people without a matched, related donor suitable for hematopoietic stem cell transplant (HSCT).
Ages: 6+ months.
Gamifant
Emapalumab-lzsg.
Approved to treat: Primary/familial hemophagocytic lymphohistiocytosis (HLH).
Ages: 0+ years old.
Report side effects/adverse events at medinfo.us@sobi.com or 866-773-5274.
Joenja
Leniolisib.
Approved to treat: Activated PI3K delta syndrome (APDS).
Ages: 12+
Report side effects/adverse events at safetyUS@pharming.com or +1-800-930-5221.
