Diagnosing and treating gliomatosis cerebri
What is a biopsy? A biopsy is an extraction of little samples of tissue that are to be checked under the microscope by pathologists. Tumors are biopsied in their origin, to assure that tumor cells are
What causes Amyotrophic Lateral Sclerosis (ALS)?
Amyotrophic lateral sclerosis: a patient’s journey «It was a sunny Sunday morning in the middle of September. Amy was playing in the local playground under the watchful eye of her parents, trying to
RareHacks: uniting experts to build the future of the rare disease community
What is RareHacks? RareHacks is a hackathon event that brings you the opportunity to apply data science tools, including machine learning and natural language processing algorithms, to a real
The POWER-tool: a new effective method that helps involving patients in clinical trial design
Research on rare diseases is often difficult to perform mainly because there are only a small number of cases to study, resulting in a vaguely representative statistical analysis of the data. For
Cancer therapies assessment indicators need to be reviewed according to a novel study
A recent publication on JAMA Internal Medicine refutes any association between progression-free survival (PFS) and health-related quality of life ( HRQoL) in cancer patients. In other words, delaying
How to guarantee quality in translational research for neuromuscular diseases
It is not easy to go from basic drug research to clinical trials on humans, and for rare neuromuscular diseases the scenario is even harder. Besides, not always all the projects arrive to the human