rare disease clinical trials

R&D

Yellow origami boat sailing a different path than a line of white origami boats

Webinar recap: The future of cell and gene therapy in rare d...

For decades, treatment options for rare diseases have been limited, often focusing on symptom management rather than addressing the root cause of them

R&D

On a journey to diverse, inclusive, and more efficient rare ...

Although orphan drugs represent a sizeable portion of new drug approvals,1most rare diseases still lack effective treatment.

R&D

Jonah Comstock interviews Dr Dhaval Shah at AAPS

Modelling biologics: Challenges and opportunities, with Dr D...

From AAPS 2025, an interview with Dr Dhaval Shah, professor of pharmaceutical sciences at the University of Buffalo and former Pfizer researcher.

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Market Access

Bridging research and clinical guidelines – interactive stra...

Medical affairs is evolving. Understanding how to harness real-world data and digital insights is essential.

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rare disease clinical trials

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