Epidermolysis Bullosa

Epidermolysis bullosa is a genetic skin condition marked by severe skin sensitivity and the formation of blisters even from minor friction or injury. This disorder arises from genetic changes that impact the proteins crucial for upholding the skin’s structural strength. The degree of severity in epidermolysis bullo...

Cytokinetics Announces Positive Results From SEQUOIA-HCM, the Pivotal Phase III Clinical Trial of Aficamten in Patients With Obstructive Hypertrophic Cardiomyopathy Cytokinetics, Incorporated released favorable top-line findings from the SEQUOIA-HCM trial (Safety, Efficacy, and Quantitative Understanding of Obst...

In recent years, the development of cell and gene therapies has provided a new avenue for the treatment of rare disorders. This article will discuss the current and future scenarios of cell and gene therapies in rare disorders with cancer indications as an exception, in Japan. Cell therapy is a treatment in which l...

Novartis Announces the Positive Results of Phase III NATALEE trial Evaluating Kisqali Novartis announced positive topline results from an interim analysis of NATALEE, a Phase III trial evaluating Kisqali® (ribociclib) plus endocrine therapy (ET) in a broad population of patients with hormone receptor-positive/hu...

Epidermolysis bullosa (EB) places a significant socioeconomic burden on patients. Several reports stated that Epidermolysis bullosa had a considerable negative impact on the quality of the lives of patients. Although it is a rare disease, EB is a cause of significant morbidity and mortality among the affected indiv...