by Peter Ciszewski | Feb 12, 2021
The U.S. Food and Drug Administration (FDA) has approved Evkeeza (evinacumab-dgnb) to treat persons 12 years and older with homozygous familial hypercholesterolemia (HoFH). Evinacumab-dgnb is a monoclonal antibody that binds to and blocks the function of...
by Peter Ciszewski | Feb 12, 2021
Lance Leopold, MD, Group Vice President, Immuno-Oncology, at Incyte, discusses retifanlimab, an investigational treatment for squamous cell carcinoma of the anal canal (SCAC). SCAC is a rare cancer in which malignant cells form in the cells that line the anus....
by Peter Ciszewski | Feb 10, 2021
John Cataldo, a 29-year old who was diagnosed with primary hyperoxaluria type 1 (PH1) at age 4, describes what it was like growing up with a rare disease. PH1 is a rare genetic disease in which excessive oxalate production leads to painful and recurrent kidney...
by Peter Ciszewski | Feb 8, 2021
Ron Cooper, President and CEO of Albireo Pharma, discusses the effect the Orphan Drug Act has had on rare disease pharmaceutical companies. As Mr. Cooper explains, the Orphan Drug Act of 1983, which facilitated the development of orphan drugs in the United...
by Peter Ciszewski | Feb 5, 2021
Auris Huen, MD, PharmD, Assistant Professor, MD Anderson Cancer Center, Department of Dermatology provides an overview of Sézary Syndrome, a subtype of Cutaneous T-cell lymphoma (CTCL). Cutaneous T-cell lymphoma belongs to the non-Hodgkin lymphoma class of...
