Rare Disease Day

R&D

On incentivisation and investment in rare disease R&D

To mark Rare Disease Day, pharmaphorum spoke with Paola Pozzi, partner of telethon strategy at Sofinnova Partners, about the outlook for investment in this field of research.

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

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R&D

Exploring the future: Opportunities and challenges in cell a...

Rare disease treatment poses distinct challenges for physicians, patients, caregivers, and pharma companies.

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