Patricia Inácio, PhD, science writer —

Amicus Therapeutics, a company developing therapeutics for rare diseases, recently released an update of the company’s clinical trial testing SD-101 as a potential therapy for patients with epidermolysis bullosa. The company’s investigational drug SD-101 is a skin cream designed for the treatment of skin blistering and lesions, and it…

Many adults and children with epidermolysis bullosa simplex (EBS-l) experience pain that can significantly impact their quality of life, but they lack access to adequate pain-management options, a questionnaire-based study found. The study, “Pain and quality of life evaluation in patients with localized epidermolysis bullosa simplex,” was published in…

The first patient has enrolled in a clinical trial evaluating both the efficacy and safety of diacerein 1% ointment (CCP-020) in treating epidermolysis bullosa simplex (EBS), Castle Creek Pharmaceuticals recently announced. The company’s Phase 2/3 DELIVERS clinical trial (NCT03154333) is currently recruiting participants. CCP-020 is a small molecule, engineered to…