There are many unmet needs in medical dermatology, especially in rare dermatologic diseases that may not have any approved therapies. In many of these cases, oral therapies that are often used off-label have demonstrated efficacy but may be associated with serious systemic toxicity, which then limits their viability as a treatment option.
Recent research has demonstrated that novel topical formulations of these drugs may represent safer treatment options that can lead to important clinically meaningful outcomes in many rare dermatologic diseases.
We are initially focused on developing new topical treatments for three rare dermatologic diseases:
Congenital Ichthyosis (CI) is a group of rare genetic keratinization disorders that leads to dry, thickened, and scaling skin.
Tuberous Sclerosis Complex
Tuberous Sclerosis Complex (TSC) is a multisystem genetic disorder that results in the growth of hamartomas.