Industry Insights

Cell and gene therapies: Where are we now?

By CONTRIBUTOR

31 July 2024

Letters

Sarcoma Awareness Month: Sarcomas demand urgent awareness and education to prevent poor outcomes

By CONTRIBUTOR

24 July 2024

Turning the tide for rare disease

I am Celine Dion: The stiff person syndrome community reacts to the release of Dion’s documentary

By CONTRIBUTOR

22 July 2024

Industry Insights

Early access to medicines: A picture is worth a thousand words

By CONTRIBUTOR

17 July 2024

Charity & advocacy

Bridging gaps in care: How independent charitable patient assistance organisations support underserved populations

By CONTRIBUTOR

10 July 2024

Industry Insights

What can rare disease services learn from oncology services? A personal reflection

By CONTRIBUTOR

1 July 2024

Letters

“Glass siblings”: an unnecessary label

By CONTRIBUTOR

24 June 2024

Turning the tide for rare disease

The UK General Election 2024: Ensuring rare diseases remain a priority

By CONTRIBUTOR

24 June 2024

RARE caregiving

A road less travelled is no less worthy: A RARE mum’s reflections on special needs parenting

By CONTRIBUTOR

21 June 2024

Reviews

Madame Web: Superheroes don’t always wear capes

By CONTRIBUTOR

18 June 2024

Charity & advocacy

A day in the life: Being diagnosed with Fanconi anaemia as a child and as an adult—what are the differences?

By CONTRIBUTOR

18 June 2024

Turning the tide for rare disease

Accelerating treatment access in the United States through financial assistance, education and advocacy

By CONTRIBUTOR

6 June 2024

Charity & advocacy

Supercharging advocacy in Latin America for Hirschsprung’s disease and anorectal malformation

By CONTRIBUTOR

31 May 2024

Letters

When children take their first breath, their care is fully funded. Why isn’t it when they take their last?

By CONTRIBUTOR

31 May 2024

Charity & advocacy

Huntington’s disease (HD) was not part of my plans, but it sure has a plan for me

By CONTRIBUTOR

29 May 2024

Turning the tide for rare disease

A rare dad’s journey: Inspiring change for the rare disease community in Rwanda

By CONTRIBUTOR

22 May 2024

Patient voice

How CLN2 Batten disease changed our life: “A Mother’s Love”

By CONTRIBUTOR

16 May 2024