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A day in the life

A day in the life: living with dyskeratosis congenita (DC)—Paula’s story

By admin
19 April 2024

Turning the tide for rare disease

Jenny Decker attempts to sail solo around the globe to raise awareness of Charcot-Marie-Tooth disease

By admin
15 April 2024

RARE News

DREAMS: a new Horizon Europe project brings hope to rare disease patients

By admin
12 April 2024

Industry Insights

As we enter a new financial year, how does the UK Budget affect the rare disease community?

By admin
10 April 2024

RARE News

Multidisciplinary expert panel explores the necessity to “shed light” on rare and undiagnosed diseases: an invitation to attend a live event at Harvard Medical School, in-person or virtually

By admin
10 April 2024

RARE News

AKU Society launches Global Patient Registry to drive research into ultra-rare disease alkaptonuria

By admin
10 April 2024

RARE News

New report: If Only You Knew

By admin
10 April 2024

RARE News

Nxera Pharma joins the World Orphan Drug Alliance

By admin
9 April 2024

RARE News

Eurordis to host 12th European Conference on Rare Diseases and Orphan Products (ECRD 2024)

By admin
4 April 2024

RARE News

Miami community comes together for CureDuchenne, raising nearly $2.5M since 2021

By admin
3 April 2024

RARE News

SMA Europe launches its 12th call for research

By admin
3 April 2024

RARE News

Foetal neonatal alloimmune thrombocytopenia (FNAIT): Fast track designation for investigational therapy to reduce the risk in alloimmunised pregnant adults

By admin
1 April 2024

RARE News

Sickle Cell Disease Association of America promotes Kevin Amado Jr.

By admin
27 March 2024

Letters to the editor

How can we get the patient heard by the regulators?

By admin
27 March 2024

RARE News

FDA approves nonsteroidal treatment for Duchenne muscular dystrophy

By admin
26 March 2024

RARE REV-inar

Eight challenges in developing rare disease therapies

By admin
25 March 2024

RARE News

Rare Disease Day 2024 – Call for greater awareness of X-linked adrenoleukodystrophy and cerebral adrenoleukodystrophy

By admin
20 March 2024
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