See all the latest news, blogs and updates from the medical sector.
News
COVID-19 vaccination for children and young people aged 12-17 years
It is recognised that a small number of children and young people aged 12-17 years with specific underlying health conditions may be at...
30/07/2021
Condition
NICE unfortunately leans towards a no for the treatment of Metachromatic Leukodystrophy (MLD).
The drug decision-making body NICE (National Institute of Clinical Excellence) has publicly released its interim decision today (9 July...
13/07/2021
News
Willow Foundation Special Days and Treats
For people living with a life-threatening illness, every day is precious. The pressures of diagnosis, treatment and recovery can have a...
08/07/2021
News
APPG on Newborn screening
On 9 June 2021, we attended a virtual APPG (All Party Parliamentary Group) meeting on Rare, Genetic and Undiagnosed Conditions about...
29/06/2021
Condition
Vimizim update from NICE for MPS IVA (Morquio)
Update 10 June 2021: Dear stakeholders, BioMarin has requested an extension to the timelines for the re-evaluation for elosulfase alfa...
03/06/2021
Blog
At home self-infusions - Carers Week 2021
In early June, the #CarersWeek campaign is celebrated annually online to bring awareness to challenges unpaid carers face and to...
03/06/2021
Condition
New COHERE study for MPS II patients
A study is currently being undertaken by the COHERE team at the Royal Manchester Children’s Hospital. ‘COHERE’ is an abbreviation of Core...
27/05/2021
Condition
Phase I/II clinical study to explore the potential of ST-920 investigational gene therapy to treat
Below is a patient newsletter from biotechnology company Sangamo about their Fabry gene therapy trial. The STAAR Study is Recruiting...
25/05/2021
News
MPS Society's chance to win £1,000
MPS Society are asking for nominations to win a £1,000 Movement for Good award from Ecclesiastical Insurance Group. MPS Society is...
24/05/2021
Story
Lexie's story
Lexie's sister Tillie Mae has MPS IIIA. Here, she shares her story about what it's like growing up with a sister who has a rare disease....
