Dystrophic Epidermolysis Bullosa
European Medicines Agency recommends ‘landmark’ gene therapy for epidermolysis bullosa
The European Medicines Agency’s Committee for Medicinal Products for Human Use recommended the approval of Vyjuvek for the treatment of wounds in patients with dystrophic epidermolysis bullosa, Krystal Biotech announced in a press release.
EMA stalls B-VEC marketing authorization for dystrophic epidermolysis bullosa
The European Medicines Agency’s Committee for Medicinal Products for Human Use cancelled the oral explanation for beremagene geperpavec-svdt’s marketing authorization application, Krystal Biotech announced in a press release.
Pregabalin improves recessive dystrophic epidermolysis bullosa pain
Patients with recessive dystrophic epidermolysis bullosa experienced a reduction in pain and itch with pregabalin treatment, according to small study.
Recessive dystrophic epidermolysis bullosa subtypes linked to type VII protein function
Based on a variant’s impact on type VII collagen protein function, patients with recessive dystrophic epidermolysis bullosa can be categorized into clinically meaningful subgroups to better guide disease management, according to a study.
FDA denies Abeona’s recessive dystrophic epidermolysis bullosa treatment
The FDA did not approve Abeona Therapeutics’ biologics license application for prademagene zamikeracel for recessive dystrophic epidermolysis bullosa, the company announced in a press release.
Researchers predict ‘friction’ between payers, physicians, patients over cost of B-VEC
The FDA’s broad indication for the use of beremagene geperpavec therapy for the treatment of dystrophic epidermolysis bullosa may have large financial implications for payers, according to a study.
Topical gene therapy improves ocular complications of dystrophic epidermolysis bullosa
Ophthalmic application of beremagene geperpavec was successful in treating recurrent cicatrizing conjunctivitis in one patient with dystrophic epidermolysis bullosa, according to a study.
CMS issues permanent J-code for Vyjuvek
Vyjuvek, the first FDA-approved treatment for dystrophic epidermolysis bullosa, has received a permanent J-code from the Centers for Medicare and Medicaid Services, Krystal Biotech announced in a press release.
Vyjuvek receives orphan drug designation in Japan for epidermolysis bullosa
The Japanese Ministry of Health, Labour and Welfare has granted Vyjuvek an orphan drug designation for the treatment of dystrophic epidermolysis bullosa, Krystal Biotech announced in a press release.
Sloughing of esophageal lining in children with dystrophic EB needs immediate expert care
The management of dystrophic epidermolysis bullosa — the most severe type of this rare condition — requires a timely clinical response and life-saving procedures, according to a study.