PEA RIDGE – Kyler Kasper is living on borrowed blood. He has not yet celebrated his second birthday, but Kyler’s parents expect him to reach it, thanks to blood donations from strangers. Kyler was recently diagnosed with Diamond Blackfan anemia, a rare blood disorder that affects about 500 people in...
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Brussels, Belgium – Patients are central to the rare disease community on all levels. This sentiment was echoed by representatives from all stakeholder groups at the 2023 World Orphan Drugs Congress which took place in early November 2023 in Barcelona. The Parliament joined the Alexion – AstraZeneca Rare Disease team...
SOUTH SAN FRANCISCO, Calif. – Kezar Life Sciences, Inc. (Nasdaq: KZR), a clinical-stage biotechnology company discovering and developing breakthrough treatments for immune-mediated and oncologic disorders, today announced the appointment of Micki Klearman, M.D. to its Board of Directors. “Micki’s addition to the board is instrumental as we continue to evaluate the...
For more than two years, Tobey Miller hasn’t been able to read her baby bedtime stories and kiss him good night. The only home he has ever known has been a room at Cardinal Glennon Children’s Medical Center, more than two hours away from her. A rare kidney disease —...
ESCONDIDO — A five-year-old kindergarten student at Pioneer Elementary School is recovering from pertussis, or whooping cough, health officials said Tuesday. The child developed the bacterial disease even after receiving the recommended five-part course of vaccinations, officials said. County Public Heath Officer Wilma Wooten explained that while the whooping cough...
NORD is excited to be hosting a series of in-person and virtual community listening sessions exclusively for Latino/a/x people affected by rare diseases in the United States. We invite you to help spread the word about this important opportunity by sharing this programming across your network and encouraging participation amongst...
Utrecht, The Netherlands and Philadelphia — LAVA Therapeutics N.V. (Nasdaq: LVTX), a clinical-stage immuno-oncology company focused on developing its proprietary Gammabody™ platform of bispecific gamma-delta T cell engagers, announced the appointment of Christy J. Oliger to its board of directors effective March 9, 2023. Ms. Oliger brings extensive commercial, portfolio...
CAMBRIDGE, Mass. – Tessera Therapeutics, a biotechnology company pioneering a new approach in genetic medicine known as Gene Writing, announced today the appointment of Howard Liang, Ph.D., as President and Chief Financial Officer. The company also expanded its executive bench with newly promoted talent and hires: Madhusudan Peshwa, Ph.D., as Chief Technology Officer for...
Leena Bruckner-Tuderman from the Department of Dermatology at the University Medical Centre in Freiburg has just been awarded the Eva Luise Köhler Research Prize for Rare Diseases. She has not only made a decisive contribution to the clarification of the molecular causes of the skin disease “dystrophic epidermolysis bullosa”, but...
Klosterneuburg, Austria – At school, he was inspired by Schrödinger´s book ‘What is Life?’. Now, several decades later, Leonid Sazanov is awarded the Erwin Schrödinger Prize by the Austrian Academy of Sciences (ÖAW). The scientist heads a research group at the Institute of Science and Technology Austria (ISTA) and was...