Medical

Medical

Transitioning successfully from paediatric to adult care in Duchenne muscular dystrophy

By admin

6 September 2023

Medical

The GLISTEN trial

By Rebecca Pender

14 April 2023

Medical

CATNAP® registry: collecting real-world data to enhance understanding of paediatric narcolepsy

By Rebecca Pender

17 November 2022

Medical

Sanius Health’s mission to personalise care for people with sickle cell disease—using advanced wearable technology

By Rebecca Pender

29 September 2022

Medical

The RAPSODI study: an online study to understand the association between the Gaucher-causing gene and Parkinson’s

By Rebecca Pender

14 September 2022

Medical

Intrahepatic cholestasis of pregnancy: a rare liver disorder that causes risk to unborn babies—the unique insights of Dr Laura Bonebrake, physician and ICP mother

By Rebecca Pender

29 June 2022

Medical

Inozyme Pharma: developing therapies for abnormal mineralisation disorders

By Rebecca Pender

9 June 2022

Medical

Seeing Red

By Rebecca Pender

26 November 2021

Medical

Zebras do exist: the diagnostic odyssey of rare disease patients

By Rebecca Pender

13 July 2021

Medical

Phase 3 ‘Proof-HD’ trial begins in Europe for people with Huntington disease

By Rebecca Pender

28 January 2021

Medical

UK’s largest ever NHS rare disease diagnosis programme launched by Mendelian and Modality NHS Partnership

By Rebecca Pender

17 December 2020

Medical

Anavex Life Sciences is the company to learn from according to the rare disease advocacy community

By Rebecca Pender

16 December 2020

Medical

Alan Finglas, founder of MSD Action Foundation and Saving Dylan, contemplates EU policy and the potential it has to open the gates for real change in the rare disease community

By Rebecca Pender

28 July 2020

Medical

Why the COVID-19 pandemic may be particularly challenging for families living with rare conditions

By Rebecca Pender

17 July 2020

Medical

My reflections on COVID-19

By Rebecca Pender

10 July 2020

Medical

Danny’s Dose changing rules and saving lives

By Rebecca Pender

12 June 2020

Medical

With these feet? – A spotlight on young onset Parkinson’s disease

By Rebecca Pender

14 April 2020

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