Charity & advocacy

Fighting for Jackson: A mother’s journey to empower families living with Angelman syndrome

By admin

13 June 2025

Charity & advocacy

The rare disease foundation journey

By admin

11 June 2025

Turning the tide for rare disease

A movement born from silence: The global mission to treat CTNNB1 syndrome

By admin

11 June 2025

Science & tech

Three ways AI is changing paediatric genomic medicine

By admin

9 June 2025

Reviews

“Nothing Solid” – a ‘vom-com’, coming soon

By admin

6 June 2025

RARE caregiving

From isolation to connection: One rare caregiver’s passion to build community

By admin

5 June 2025

Patient voice

“You look different.” 

By admin

4 June 2025

Charity & advocacy

Building a global patient registry for a rare disease: The story of the INPDR

By admin

2 June 2025

RARE News

NephCure celebrates inaugural FSGS Awareness Day on June 10, 2025

By admin

29 May 2025

RARE News

CureDuchenne Ventures invests in Entos Pharmaceuticals for development of a redosable, full-length dystrophin gene therapy

By admin

29 May 2025

Turning the tide for rare disease

A tapestry of hope, advocacy & community: Day 2 of the NPUK AFC and IW 2025 (Part 1)

By admin

26 May 2025

Reviews

Blackbird Lets His Papers Fly

By admin

26 May 2025

Reviews

Lou Gehrig’s prayers

By admin

26 May 2025

Reviews

The seventh age of Shakespeare’s father

By admin

26 May 2025

Reviews

They asked, again for DNA

By admin

26 May 2025

Turning the tide for rare disease

Highlighting breakthroughs and collaboration: The NPUK Annual Family Conference & Interactive Workshop 2025

By admin

26 May 2025

Reviews

Carpenter’s Rest, A Lullaby

By admin

26 May 2025