R&D AI could provide breakthrough for early diagnosis of rare di... For people living with rare diseases, one of the major challenges is being able to receive a rapid and accurate diagnosis.
R&D Sponsored Trials and tribulations of drug development: What does it ta... Rare diseases are a complex and challenging area of medicine, affecting millions of people worldwide.
R&D Making accessibility strategy part of your rare disease tria... Over the past several years, diversity, equity, and inclusion (DEI) has become a central subject of conversation in clinical trials.
Patients Challenges and opportunities with plasma-derived therapies This newest pharmaphorum podcast welcomes Takeda's Dr Kristina Allikmets.
R&D Sponsored Trials and tribulations of drug development What does it take to establish new treatments for rare diseases?
News Hemab raises $135m for rare bleeding disorder trials Cash will be used to advance drugs for Glanzmann thrombasthenia and von Willebrand disease.
News Neurogene drops high-dose arm in Rett study after death The patient affected by a serious adverse event in Neurogene's clinical trial of Rett syndrome gene therapy has now died
Sales & Marketing Coming soon: The Life Sciences Industry Report 2025 – insigh... Prepare for 2025 with pharmaphorum’s Life Sciences Report, featuring expert insights on AI in drug development, gene therapy, and top industry trends
