I once told a conference that it was my perception that rare disease communities were in a stronger position to put research at the top of the agenda; that patients, carers, clinicians and researchers found it easier to get around the table in these areas, were able to work more closely together and mobilise for … Continue reading Understanding why people with rare diseases lose out on research may help those with other more common conditions @rarediseaseday
Category: genomes
Cystic Fibrosis Gene Therapy Trial testament to the cf community as a positive, patient and persistent partner in research
I hope you will have seen or heard the news today of the positive results of a gene therapy trial for people with cystic fibrosis. And this is how The Guardian is reporting it. Although it is early days and the improvement shown in patients only a modest one, it is nonetheless a very hopeful … Continue reading Cystic Fibrosis Gene Therapy Trial testament to the cf community as a positive, patient and persistent partner in research
