Turning the tide for rare disease

Rare Land: a Greek film shining a light on alpha mannosidosis and rare disease

By Rebecca Pender

12 December 2022

Press releases

CureDuchenne raises more than $1.5 million at eighth annual Napa in Newport to help find a cure for Duchenne muscular dystrophy

By Rebecca Pender

8 December 2022

Press releases

NFL players spotlight Sickle Cell Disease Association of America

By Rebecca Pender

7 December 2022

Charity & advocacy

#DuchenneCan: celebrating what people with Duchenne CAN achieve

By Rebecca Pender

5 December 2022

Turning the tide for rare disease

Matt Hay: becoming a neurofibromatosis advocate and giving back to his community

By Rebecca Pender

1 December 2022

Turning the tide for rare disease

Jessica Massengale: living strong with scleroderma

By Rebecca Pender

24 November 2022

Turning the tide for rare disease

A celebration of 25 years in advocacy: Tanya Collin-Histed, CEO of the International Gaucher Alliance

By Rebecca Pender

18 November 2022

Medical

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

By Rebecca Pender

17 November 2022

Charity partner news

DEBRA launches £5m fundraising appeal to stop the pain of EB

By Rebecca Pender

16 November 2022

Press releases

Winners of Gene People Awards 2022 announced

By Rebecca Pender

16 November 2022

RARE REV-inar

Early access pathways to medicines – insights from a multi-stakeholder discussion

By Rebecca Pender

10 November 2022

Turning the tide for rare disease

Finding a cure nobody else is looking for: why Geri and Zach shoot for the moon

By Rebecca Pender

31 October 2022

Science & tech

The real-world evidence revolution: how better research can improve patients’ lives

By Rebecca Pender

27 October 2022

Charity partner news

Leading Muscular Dystrophy charity gives petrol heads a ‘driving experience of a lifetime’ as it continues its commitment to delivering life-changing wish-fulfilment experiences to children and young adults

By Rebecca Pender

26 October 2022

Press releases

CureDuchenne launches Occupational therapist certification programme to improve care for individuals with Duchenne muscular dystrophy

By Rebecca Pender

20 October 2022

Charity partner news

The Student Voice Prize: an international essay competition raising the profile of rare disease

By Rebecca Pender

13 October 2022

Turning the tide for rare disease

What I learned raising a son with haemophilia: Stormy’s story

By Rebecca Pender

12 October 2022