Congenital myopathies – centronuclear myopathies

Clinical trial designs in rare and ultra-rare diseases yield multiple challenges due to restricted sample sizes. In the context of an ongoing investigation of centronuclear myopathies (CNMs), a group of congenital neuromuscular diseases whose most severe and most common incident form is X-linked, affecting approximately 1 in 50,000 newborn boys, this feature holds especially true. In such settings, innovative Bayesian methods can often pay dividends, allowing the sensible incorporation of auxiliary data and other relevant information to bolster that collected by the trial itself.
Source: Neuromuscular Disorders - Category: Neurology Authors: Source Type: research