Articles by tag: orphan-drug

Targeted therapy for Usher syndrome 1B: new data on AAVB-081

Interim data from phase 1/2 gene therapy AAVB-081 for Usher syndrome 1B: safety, efficacy, and prognosis. Learn about the dual AAV vector and treatment prospects.

FDA approved bulevirtide (Hepcludex): treatment of hepatitis delta in the USA

FDA approved the first US drug for hepatitis delta — bulevirtide (Hepcludex). Learn about dosage, risks, price, and prognosis. Read the full analysis.

Bulevirtide (Hepcludex®): FDA approval for hepatitis D

FDA approved the first drug for chronic hepatitis D — bulevirtide. Mechanism, doses, risks, price, and prognosis. Find out who is suitable for therapy.

Enspryng reduced the risk of relapses in MOGAD by 68%

Genentech proved the efficacy of satralizumab in a rare autoimmune disease. The risk of attacks decreased by 68%, new lesions by 79%. Learn how the first targeted drug fills the niche of empirical therapy.