Medical

Medical

Systemic mastocytosis: A new study reveals the perceptions and realities of this rare mast cell disorder

By CONTRIBUTOR

2 October 2024

Medical

Goal Attainment Scaling: delivering patient-centred clinical trials using personalised outcome measures

By Geoff Case

11 September 2024

Medical

Transitioning successfully from paediatric to adult care in Duchenne muscular dystrophy

By CONTRIBUTOR

6 September 2023

Medical

The GLISTEN trial

By CONTRIBUTOR

14 April 2023

Medical

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

By CONTRIBUTOR

17 November 2022

Medical

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

By CONTRIBUTOR

29 September 2022

Medical

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

By CONTRIBUTOR

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 CONTRIBUTOR

29 June 2022

Medical

Inozyme Pharma: developing therapies for abnormal mineralisation disorders

By CONTRIBUTOR

9 June 2022

Medical

What can we learn from European rare disease policies to future-proof our health systems?

By CONTRIBUTOR

4 April 2022

Medical

Seeing Red

By CONTRIBUTOR

26 November 2021

Medical

Zebras do exist: the diagnostic odyssey of rare disease patients

By CONTRIBUTOR

13 July 2021

Medical

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

By CONTRIBUTOR

28 January 2021

Medical

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

By CONTRIBUTOR

17 December 2020

Medical

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

By CONTRIBUTOR

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 CONTRIBUTOR

28 July 2020

Medical

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

By CONTRIBUTOR

17 July 2020

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