by Madaline Spencer | Aug 12, 2025
Tobias Hagedorn, Secretary of the European Society for PKU (ESPKU), discusses unmet medical needs in rare disease communities. Phenylketonuria (PKU) is a genetic metabolic disorder that increases the body’s levels of phenylalanine, a natural amino...
by Madaline Spencer | Aug 11, 2025
The U.S. Food and Drug Administration (FDA) has approved Modeyso (dordaviprone) for the treatment of patients with diffuse midline glioma with a H3 K27M mutation. Glial tumors are a type of brain tumor originating in glial cells. Diffuse midline H3 K27M-mutant glioma...
by Madaline Spencer | Aug 11, 2025
Dirk Arnold, MD, PhD, Director of the Asklepios Tumorzentrum Hamburg, discusses results from the OrigAMI-1 clinical trial in left-sided RAS/BRAF wild-type metastatic colorectal cancer (mCRC). Approximately 50% of patients diagnosed with colorectal cancer...
by Madaline Spencer | Aug 10, 2025
Skand Shekhar, MD, Principal Investigator at the National Institutes of Health (NIH), discusses Erdheim-Chester disease diagnosis and treatment options. ECD is a rare blood cancer characterized by the overproduction of histiocytes, which then accumulate...
by Madaline Spencer | Aug 8, 2025
The U.S. Food and Drug Administration (FDA) has approved Alhemo (concizumab) injection as a once-daily prophylaxis treatment to prevent or reduce frequency of bleeding episodes in patients ages 12 years and older with hemophilia A or B without inhibitors. This is an...
