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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

Industry Insights

Rare Disease Day 2024: Sheela Upadhyaya previews some of the events taking place across the world

By admin
14 February 2024

Industry Insights

The UK Rare Diseases Framework: Sheela Upadhyaya reflects on recent progress across the UK

By admin
12 February 2024

Patient voice

Love isn’t rare—Ken To’s journey with Huntington’s disease

By admin
11 February 2024

Press releases

Amylyx Pharmaceuticals announces completion of enrollment in HELIOS, a Phase 2 Study of AMX0035 for the Treatment of Wolfram Syndrome

By admin
9 February 2024

Press releases

MEF2C Foundation & MUSC launch program to develop MEF2C therapeutics

By admin
9 February 2024

Turning the tide for rare disease

Congenital muscular dystrophy: Kelly and Avery’s mission to challenge perceptions of disability

By admin
7 February 2024

Press releases

National Tay-Sachs & Allied Diseases Association hosts first of its kind drug development meeting for GM2

By admin
2 February 2024

Press releases

Get Ready to #ShowYourStripes for Rare Disease Day 2024 29th February: the rarest day of the year!

By admin
1 February 2024
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