See all the latest news, blogs and updates from the medical sector.
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...
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...
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...
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...
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...
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...
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...
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...
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...
Could it be Fabry? A response
Commentary by Professor Atul Mehta, formerly Director of the Lysosomal Diseases Centre, Royal Free Hospital and University College London...