Most patient organizations, no matter how small, start with the same mission: to raise awareness and funds for research in their disease. But what research are they doing? are they all starting in the same place? and do they all evolve to run the same type of research? In my experience there are many types of strategy and approaches that patient groups take, in particular when they are starting. What a patient organization means when they say “we are doing research”, therefore, hides many different possibilities. The article describes some of these types, and discusses their advantages and disadvantages.

INTRODUCTION TO THE SERIES - The field of rare diseases, where each disease affects only hundreds or thousands of patients world-wide, is leading this revolution. Their diseases were once orphan to medicine, too rare to receive sufficient attention and investment to move the needle. Today patients take ownership of their research fields, and move the needle themselves. They have become the center of the network and the expert in the room. They are impatient, not wanting to wait for pharma to take an interest in their disease. They are impatient patients and are leading an impatient revolution. 

Dravet syndrome pipeline review 2018 - Summary in Spanish for Dravet families.

Los avances en torno al tratamiento del síndrome de Dravet en los últimos años han seguido pasos agigantados. En los últimos 5 años hemos experimentado una explosión en el número de programas en desarrollo para tratar la enfermedad: de tener solo Diacomit y nada más a tener Diacomit y 14 más en desarrollo.​​​​​​​ Este texto es un resumen en Español del Dravet syndrome pipeline review 2018 dirigido a familias, con el texto no solo traducido sino también ajustado a los intereses de aquellos que tienen un hijo/a con síndrome de Dravet y su entorno.

After May 2018 patients will have the right to request access to their data files. They will have the right to withdraw their data from a database that is not being used in the way that the patient through it would operate. And they will have the right to obtain these files generated by a particular data holder, for example a gene testing lab, and get the usable file and share it with as many studies and registries as the patient wants. The EU General Data Protection Regulation gives patients the Right to Access, the Right to be Forgotten, and the Data Portability right.