To understand parents’ experiences of having a child diagnosed with CCALD
Researchers from Sheffield Hallam University are conducting research in collaboration with Alex TLC to understand parents’ experiences of having a child diagnosed with Childhood Cerebral Adrenoleukodystrophy (CCALD). The study is a first of it’s kind and will help to provide evidence towards Newborn screening for ALD. It is a valuable study with the results providing a good opportunity to improve the circumstances for other families. The results will directly feed into initiatives to improve diagnosis and access to treatments for ALD patients.
Come join us for a Coffee & Mince Pie Morning Catch Up!
Come join us for a festive coffee and mince pie morning catch up on Wednesday 15th December from 11:00am-12:00pm!
Rarely Heard by Findacure & Same But Different
A rare disease podcast series that exists to share the stories, experiences, knowledge, insights and voices of those living with rare diseases. The debut season will be focusing on mental health.
Poxel Announces the Formation of it’s Scientific Advisory Board for Rare Metabolic Diseases
Poxel are a clinical stage biopharmaceutical company developing innovative treatments for chronic diseases with metabolic pathophysiology, including non-alcoholic steatohepatitis (NASH) and rare disorders.
Our Support Services Manager interview with Pharmafocus magazine
Karen Harrison, Our Support Services Manager, did an interview with Pharmafocus magazine
By the UK Newborn Screening Committee about what should be screened for and how
Questions from the Progress Educational Trust event ‘Newborn Screening: What Should Be Screened for and How? meeting have been answered by the UK Newborn Screening Committee