See all the latest news, blogs and updates from the medical sector.
Condition
Treatment for Metachromatic Leukodystrophy (MLD) approved by the European Commission
MLD is a very rare, fatal genetic disorder caused by mutations in the ARSA gene which lead to neurological damage
22/12/2020
News
Frequently asked questions about the vaccination programme for COVID-19
The BIMDG has prepared these responses to frequently asked questions about the vaccine progamme for COVID-19.
18/12/2020
Blog
Daniella Vandepeer discusses her clinical trial experience
Daniella Vandepeer shares her experiences of family life on a clinical trial including her motivation for joining a trial, the difficulties the family faces and what she feels is important for others considering joining a clinical trial.
13/12/2020
Blog
Louise's story - The Big Give Christmas Challenge 2020
Meeting families in person has supported my learning about these conditions and my understanding about the challenges families face.
23/11/2020
News
Kelly Mills' charity appeal for MPS Society on Radio 4
Thank you so much to everyone who donated to the Radio 4 appeal after hearing Kelly talk about her daughter, Penny, and her experience of...
04/11/2020
Blog
So what does clinical trial support services actually mean? Lianne from RDRP reveals all
From clinical trials support and specialist medical communications to research and real-world evidence, Rare Disease Research Partners...
09/10/2020
Condition
AVROBIO announce collaborative research agreement with Manchester university for MPS II gene therapy
AVROBIO announce collaborative research agreement with Manchester university for MPS II gene therapy.
04/10/2020
Condition
REGENXBIO announces expansion of gene therapy programme for the treatment of MPS II
REGENXBIO have released an update on the progress of their investigational gene therapy programme for MPS II Hunters disease.
02/10/2020
Blog
Back to school - September 2020 brings mixed emotions for our families
After many months of parents across the country tasked with teaching their children from home, our families have shared their back to...
21/09/2020
News
Tune in to Kelly Mills' charity appeal for MPS Society on Radio 4 this month
Tune in to hear Kelly's appeal for MPS Society on Radio 4 this month.
