Turning the tide for rare disease

Turning the tide for rare disease

Dr Justin West’s mission to accelerate the discovery of a disease-modifying treatment for KCNT1-related epilepsy

By CONTRIBUTOR

10 June 2022

Medical

Inozyme Pharma: developing therapies for abnormal mineralisation disorders

By CONTRIBUTOR

9 June 2022

RARE News

Gene People celebrates their first anniversary!

By admin

6 June 2022

Charity & advocacy

Maryland governor proclaims June 11 is KBG syndrome awareness day

By CONTRIBUTOR

6 June 2022

Patient voice

Making peace with numbers

By CONTRIBUTOR

6 June 2022

Charity & advocacy

Let’s Play Fair—Disability Charity Scope launches accessible play campaign which demands that every child has an equal right to play

By CONTRIBUTOR

3 June 2022

Turning the tide for rare disease

A mother’s mission to raise awareness of Bardet-Biedl syndrome and promote effective partnerships between carers and medical professionals

By CONTRIBUTOR

30 May 2022

RARE News

CureDuchenne hosts conversation with Pfizer to discuss opening of first U.S. sites in phase 3 trial of investigational gene therapy for ambulatory patients with Duchenne muscular dystrophy

By admin

26 May 2022

Turning the tide for rare disease

The Vascular Birthmarks Foundation: revolutionising awareness levels, access to treatments and physician education

By CONTRIBUTOR

26 May 2022

RARE News

Lace up to raise awareness of PSP and CBD: PSPA relaunches fun campaign to raise awareness of rare brain conditions progressive supranuclear palsy (PSP) and corticobasal degeneration (CBD)

By admin

23 May 2022

RARE News

World Orphan Drug Alliance – combining forces to reach patients with rare diseases across the globe

By admin

23 May 2022

Charity & advocacy

Rare Diseases Organization Nepal holds inaugural meeting to formalise its mission, vision, values and main objectives

By CONTRIBUTOR

23 May 2022

RARE News

The World Orphan Drug Congress USA Boston July 11-13

By admin

23 May 2022

Turning the tide for rare disease

Saarah’s Foundation: a fitting legacy to Saarah Ahmed—daughter, sister, star student, aspiring neuroscientist, Miss Universe GB contestant and kEDS warrior

By CONTRIBUTOR

20 May 2022

Science & tech

Sure, you’ve heard about GNEM. But do you know about the Bulgarian variant?

By CONTRIBUTOR

19 May 2022

RARE News

GARDIAN Registry for types 2 & 3 Gaucher disease is now open for registrations

By admin

17 May 2022

Turning the tide for rare disease

The first step in advocacy is believing in the power of your own voice

By CONTRIBUTOR

16 May 2022

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