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

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 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

RARE News

Rare disease advocates learn to accelerate therapeutic development at 9th RARE Drug Development Symposium

By admin

19 March 2024

RARE News

Alnylam launches Hereditary ATTR (hATTR) Amyloidosis campaign to help shorten diagnosis time

By admin

19 March 2024

RARE News

New primer highlights latest understanding of rare inherited disease alkaptonuria

By admin

19 March 2024

RARE News

DEBRA UK asks the British public to BE the difference for EB in 2024

By admin

19 March 2024

RARE News

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

By admin

1 March 2024

RARE News

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

RARE News

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