by CheckRare Staff | Jan 9, 2024
Kim Moran, SVP, head of U.S. Rare Diseases at UCB, discusses Zilbrysq and the Phase 3 study that led to its approval. Transcription: We have the US FDA approval for ZILBRYSQ, which is based on our pivotal study, which is a Phase 3 study under the acronym of RAISE. It...
by CheckRare Staff | Jan 8, 2024
Björn Mellgård, MD, PhD, Vice President, Global Program Lead, Rare Genetics and Hematology, Takeda, discusses the steps taken for Adzynma to get FDA approved for patients with congenital thrombotic thrombocytopenic purpura (cTTP). Transcription: We had a total of 46...
by CheckRare Staff | Jan 3, 2024
Eric Crombez, MD, Chief Medical Officer of Ultragenyx, describes osteogenesis imperfecta. Transcription: I’m Eric Crombez. I’m Chief Medical Officer at Ultragenyx. I’m a biochemical geneticist by training, so I do have experience during my...
by CheckRare Staff | Jan 2, 2024
First characterized 30 years ago, aromatic L-amino acid decarboxylase (AADC) deficiency is a rare, genetic disorder that affects the central nervous systems of young patients [1,2]. AADC deficiency is caused by defect in the dopa decarboxylase (DDC) gene, which leads...
by CheckRare Staff | Dec 26, 2023
Rare genetic conditions affecting blood vessels can have severe and lifelong symptoms, including seizures and impaired development. These conditions, such as Sturge-Weber syndrome and Phakomatosis Pigmentovascularis with Dermal Melanocytosis (PPV-DM), can...
