Subscribe Now

By entering these details you are signing up to receive our newsletter.

Press releases

Launching CamRARE’s ‘The Need for a RARE Patient Passport’ Report for Rare Disease Day

By admin
1 March 2024

Press releases

Coave Therapeutics Receives Grant from the ALS Association to Advance its CTx-TFEB Program as a Potential Treatment for All Forms of ALS

By admin
29 February 2024

Press releases

SMA Europe launches “Care for adults living with SMA in Europe: a benchmarking report.”

By admin
29 February 2024

Charity & advocacy

One in a million

By admin
28 February 2024

Patient voice

Raising a child with Hirschsprung’s disease

By admin
28 February 2024

Charity & advocacy

300 million colours of Rare: three patients and caregivers share their rare disease stories

By admin
28 February 2024

Science & tech

Beyond development: Overcoming market access challenges for rare disease treatment

By admin
28 February 2024

Press releases

£1.5 million new fund launched to ‘Help the Heart’

By admin
27 February 2024

Press releases

Blueprint Medicines highlights AYVAKIT® (avapritinib) long-term efficacy and safety data and advances in mast cell disease research at 2024 AAAAI annual meeting

By admin
23 February 2024

Press releases

CureDuchenne hosts “Champions in Miami” event on March 9 to help find a cure for Duchenne muscular dystrophy

By admin
23 February 2024

Turning the tide for rare disease

Guiding Grayson: a family’s fight to advocate, educate and save their son, Grayson, from CLN3 Batten Disease

By admin
21 February 2024

Press releases

CMT Research Foundation partners to advance study of CMT type 1J by Dr Stephan Zuchner

By admin
21 February 2024

Science & tech

Dr Wendy Chung and Simons Searchlight: pioneering research into genes that cause rare neurodevelopmental disorders

By admin
21 February 2024

Press releases

Patients as Partners in Clinical Research Conference 2024

By admin
19 February 2024

A day in the life

A day in the life: living with Bardet-Biedl syndrome—Tessa’s story

By admin
16 February 2024

Charity & advocacy

Rare Disease Awareness Day Calendar

By admin
14 February 2024

A day in the life

Don’t let your condition define you! David’s journey with stiff person syndrome

By admin
14 February 2024
Skip to content