FDA places hold on Abeona late-stage study
Abeona Therapeutics announced that the FDA has placed a clinical hold on its planned Phase 3 clinical trial evaluating autologous cell therapy EB-101 in patients with recessive dystrophic epidermolysis bullosa.
Abeona Therapeutics announced that the U.S. FDA has removed its clinical hold on the company ’s pivotal Phase 3 clinical trial evaluating gene therapy for the treatment of recessive dystrophic epidermolysis bullosa (RDEB).
Conclusion: The neonate was discharged with shorter hospitalization time and with total epithelization of the lesions, despite the severity. The family actively participated in the care process, demonstrating the ability to perform the dressings at home.
Dystrophic epidermolysis bullosa (DEB) is a hereditary skin fragility disorder, characterized by trauma-induced blistering followed by soft tissue fibrosis. One of the most feared complications is the early de...
The term epidermolysis bullosa (EB) comprises a number of rare and genetically heterogenous disorders characterized by structural skin fragility that results in recurrent blister formation.1 Dystrophic epidermolysis bullosa (DEB) is a subtype of EB that is caused by mutations in the COLA1 gene encoding type VII collagen; it is characterized by blister formation in the dermis, directly beneath the epidermis, and sometimes in the mucous membranes.2 The reported incidence of DEB is 26.4 new cases per 1,000,000 live births.
Authors: Zhang J, Ding Y, Li M, Yao Z, Zhuang Y Abstract Epidermolysis bullosa simplex, generalized severe (EBS-gen sev) is one of the major forms of EBS, caused by mutations of the keratin 5 (KRT5) or keratin 14 (KRT14). However, it is rarely reported in the Chinese population. The current study was performed on three unrelated Chinese families with five patients clinically suspicious for distinct stages of EBS. Mutation screening was performed by direct sequencing of the entire coding regions of KRT5 and KRT14 genes. A diagnosis of EBS-gen sev for patients in these three families was confirmed by revealing missen...
Recessive dystrophic epidermolysis bullosa (RDEB) is a hereditary blistering disorder due to lack of type VII collagen (C7). At present, treatment is mainly supportive.
PMID: 31773727 [PubMed - as supplied by publisher]
Condition: Recessive Dystrophic Epidermolysis Bullosa Interventions: Drug: Rigosertib Sodium; Other: Quality-of-Life Assessment Sponsors: Thomas Jefferson University; Onconova Therapeutics, Inc. Not yet recruiting