Patients, Families & the Science of Rare Diseases

On the 28th February, we commemorated Rare Disease day. The majority of rare diseases affect children, with an estimated 70% having a Paediatric onset (source There is a huge, unmet need to develop effective drugs for children with rare diseases. Currently, only 5% of rare diseases have an effective treatment option.

Expert advice in pediatric drug development by c4c

Clinical, methodology, and patient/carer expert advice in pediatric drug development by conect4children – CTS Journal – Wiley Online Library   Many medicines are used “off-label” in children outside the terms of the license and feasible pediatric clinical trials are a challenge to design. Conect4children (c4c) is an Innovative Medicines Initiative project to set up a…