Research & Consultant Companies

A pivotal shift is taking shape in the landscape of Friedreich's ataxia research. With seemingly boundless potential, interferon gamma emerges as a promising beacon...

The dynamic field of orphan drug development encounters new hurdles as zeteletinib, initially granted an orphan designation for medullary thyroid carcinoma treatment, has been...

In a significant development within the field of rare disease treatment, a new therapy has been granted orphan designation by the European Medicines Agency...

In a significant stride for medical research, the European Union's Committee for Orphan Medicinal Products (COMP) has granted orphan medicine designation to a groundbreaking...

Nuvisertib has taken a significant step forward, with the European Union granting it orphan designation for the treatment of myelofibrosis. This development signifies a...

In a landmark move for the treatment landscape of rare diseases, the European Union has granted an orphan designation to Elafibranor for primary sclerosing...

The landscape of ovarian cancer treatment in Europe is poised for significant advancement following a recent announcement from the European Medicines Agency (EMA). On...

The European Medicines Agency (EMA) has recently extended orphan designation status to a new treatment aimed at combating Duchenne muscular dystrophy, a debilitating genetic...

In a pivotal move for those affected by rare diseases, the European Medicines Agency (EMA) has granted orphan designation to Luminol Monosodium. This pivotal...

In the realm of medical advancements, the European Union recently highlighted a promising development with an announcement on orphan designation for a potential therapy...

Adrenal insufficiency is a rare and potentially life-threatening condition affecting a small population in the European Union. Despite the availability of several treatments, including...

A potential treatment for CSF1R-related leukoencephalopathy, characterized by the withdrawal of its orphan designation in the European Union, highlights the dynamic nature of pharmaceutical...

In a move that underscores the persistent search for solutions to debilitating genetic disorders, the European Commission granted orphan designation to an adeno-associated viral...

Progress in rare disease treatment often requires not just scientific innovation, but also strategic regulatory support. The European Medicines Agency (EMA) recently took a...

Retinitis pigmentosa, a debilitating eye disorder leading to progressive vision loss, stands on the brink of receiving a promising genetic intervention. The condition, characterized...