Rare Disease Day

Digital

Medicine doctor writing electronic medical record

Driving innovation in rare diseases: Digital health and coll...

In recognition of Rare Disease Day, Dr Kamran Iqbal, head of medical affairs and patient advocacy in global rare diseases at Chiesi UK, discussed with pharmaphorum the company’s evolving ro

News

Clinigen educates on early access to rare disease therapies

On Rare Disease Day, Clinigen has launched a campaign to try to encourage doctors and patients to explore how they can get early access to investigational therapies.

R&D

Rare Disease Day: Enhancing clinical trial success in rare d...

Rare diseases impact an estimated 300 million people globally, while around one in five cancers are considered a rare disease.

R&D

Looking to the future of clinical trials: Gene therapy, prec...

Next-generation technologies in medicine have significantly expanded our ability to identify and address rare diseases, propelling forwards the development of more effective and targeted tr

Patients

Rare Disease Day 2024 – The importance of shining a light on...

I have spent the last 15 years working in rare diseases, working with some of the most dedicated ambassadors for rare disease, from researchers to patient advocates to patients and families

R&D

The opportunities and challenges facing rare disease therapi...

To mark upcoming Rare Disease Day, pharmaphorum asked a panel of experts to give their thoughts on the opportunities and challenges facing developers of rare disease therapies in 2024.