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.
Revcovi
Recombinant adenosine deaminase.
Approved to treat: Adenosine deaminase (ADA)-deficient severe combined immune deficiency (SCID).
Report side effects/adverse events at us.medical@chiesi.com or 1-888-661-9260.
Actimmune
Recombinant interferon gamma-1b.
Approved to treat: Chronic granulomatous disease (CGD).
Report side effects/adverse events at 1-866-479-6742 (Option 1).
Complement deficiencies
Individuals with a complement deficiency, including people with hereditary angioedema, can have clinical problems that are a result of the role that the specific complement protein plays in the normal function of the human body.
Project ECHO clinician series: Addressing social determinants of health
July 10, 2023
Watch video
NEMO deficiency syndrome
NEMO deficiency syndrome is a complex disease caused by genetic variants in the X-linked NEMO gene (also known as IKK gamma or IKKG).
Hyper IgM syndromes (HIGM)
Hyper IgM syndromes (HIGM) are characterized by decreased levels of immunoglobulin G (IgG) in the blood and normal or elevated levels of immunoglobulin M (IgM).
