Chanarin-Dorfman syndrome (CDS) is a rare autosomal recessive disorder manifesting predominantly as congenital ichthyosiform erythroderma and systemic accumulation of neutral lipids. Mutations in ...

Timber's lead candidate TMB-001 for rare skin disease congenital ichthyosis failed to meet its efficacy objectives across the board in the ASCEND trial and will not support a planned regulatory ...

Phocomelia is a rare condition present from the time of birth. In this, people are born with arms or legs that are short or not fully formed. The hands or feet may be attached directly to the baby ...