Press releases

SpringWorks Therapeutics announces FDA approval of Ogsiveo™ (nirogacestat) as the first and only treatment for adults with desmoid tumours

By admin

8 December 2023

Press releases

Worthing couple take part in cooking challenge supported by celebrity chefs to raise funds for Muscular Dystrophy

By admin

8 December 2023

Science & tech

The Undiagnosed Diseases Network’s mission to eradicate the diagnostic odyssey: an interview with Sarah Marshall

By admin

6 December 2023

A day in the life

A day in the life: a medically complex condition that is as unpredictable as the weather

By admin

29 November 2023

Charity & advocacy

Thoughts into Action: What is quality of life as defined by people living with inherited metabolic disorders?

By admin

22 November 2023

Press releases

CureDuchenne raises more than $1.3 million at ninth annual Napa in Newport to help find a cure for Duchenne muscular dystrophy

By admin

16 November 2023

Charity & advocacy

Shining a light on Smith-Magenis syndrome

By admin

15 November 2023

Science & tech

What is a flare in Sjögren’s? TREND Community’s innovative use of artificial intelligence to find answers for the community

By admin

8 November 2023

Press releases

Exciting news: $100,000 in funding for Myhre syndrome research projects

By admin

7 November 2023

Press releases

Rare Stories initiative illuminates lives in Northern Ireland rare disease community

By admin

2 November 2023

Patient voice

Fostering a positive mind and body

By admin

1 November 2023

Press releases

CureDuchenne welcomes new executive director, vice president of medical affairs, and board members 

By admin

25 October 2023

Editor's picks

Let’s talk about sex, baby!

By admin

25 October 2023

Charity & advocacy

Do you work for a compassionate employer?

By admin

18 October 2023

Industry Insights

Together4RD: making ERN-industry collaboration a key pillar of the future in the rare disease research ecosystem

By admin

11 October 2023

A day in the life

A day in the life: navigating our way through the labyrinth of a newly discovered disease

By admin

9 October 2023

Press releases

Arthur Suckow, former co-founder and chief executive officer of DTx Pharma, joins CMT Research Foundation board of directors

By admin

5 October 2023