The outlook for drug development to treat rare diseases looks encouraging, offering hope to the 300 million people globally facing the significant challenges of these often-overlooked condi
Recruiting patients for rare disease trials is akin to finding a needle in a haystack, with challenges such as geographical dispersion, low awareness, and limited eligible patient pools.
The FDA has said it will create a rare disease innovation hub to help speed the development of new treatments and build connections between developers and the rare disease community.
The last three slots for projects that will be included in the FDA’s new programme to accelerate the development of therapies for rare diseases have been taken by Moderna,
In a new white paper from the World Without Disease initiative, a 2024 update is provided of the current endeavours and developments that have occurred since inaugural discussions last year