Turning the tide for rare disease

Libmeldy gene therapy offers a lifeline to children with leukodystrophy and gives hope to the rare disease community

By CONTRIBUTOR

23 March 2022

Turning the tide for rare disease

Turning the tide for adult polyglucosan body disease (APBD)

By CONTRIBUTOR

16 March 2022

Turning the tide for rare disease

The Global Genes Rare Disease Diversity, Equity and Inclusion Report: the challenges

By CONTRIBUTOR

16 March 2022

Turning the tide for rare disease

The healing power of storytelling

By CONTRIBUTOR

14 March 2022

Turning the tide for rare disease

Gillian Jackson: bringing positive energy and information to the online BBS community

By CONTRIBUTOR

11 March 2022

Charity & advocacy

The Vascular Birthmarks Foundation, Dr. Giacomo Colletti, Laserplast, and Candela collaborate to provide pro bono laser treatments

By CONTRIBUTOR

10 March 2022

Turning the tide for rare disease

How 10,000 people living with disability could end the foster care crisis

By CONTRIBUTOR

7 March 2022

Turning the tide for rare disease

Brace! Brace! Brace… for burnout!

By CONTRIBUTOR

2 March 2022

Turning the tide for rare disease

Turning the tide for rare disease

By CONTRIBUTOR

28 February 2022

Industry Insights

Living with Gaucher disease: challenging encounters along the patient journey

By CONTRIBUTOR

17 February 2022

Patient voice

Cavernomas—a patient’s efforts to understand the possible systemic implications of a defect in the KRIT1 (CCM1) gene

By CONTRIBUTOR

14 February 2022

Patient voice

The global disability community and the DE&I discussion: a match that needs to be made

By CONTRIBUTOR

9 February 2022

Patient voice

Everyone, on the bus!

By CONTRIBUTOR

28 January 2022

A day in the life

My year at RARE Youth Revolution

By CONTRIBUTOR

26 January 2022

Charity & advocacy

Living with muscular dystrophy in Nepal

By CONTRIBUTOR

13 December 2021

Patient voice

Caring for his children with VICI syndrome: Muzaffar Anjum’s story

By CONTRIBUTOR

8 December 2021

Patient voice

“Do you hear me?”

By CONTRIBUTOR

3 December 2021