On Biostatistics and Clinical Trials
CQ's web blog on the issues in biostatistics and clinical trials.
Showing posts with label
Rare diseases
.
Show all posts
Showing posts with label
Rare diseases
.
Show all posts
Wednesday, May 17, 2023
Another successful trial with randomized withdrawal design
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Biotech company PTC Therapeutics announced today that their phase III study of Sepiapterin in PKU patients achieved the primary efficacy en...
Thursday, June 09, 2022
Drug Development for Rare Diseases - Public Workshops
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Drug development for rare diseases is challenging, complex, but absolutely necessary. FDA has special programs to manage and encourage the d...
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