Zolgensma (onasemnogene abeparvovec), Novartis’ gene therapy for spinal muscular atrophy (SMA), recently announced landmark long-term follow-up data that children treated with Zolgensma were able to maintain key motor functions after 10 years, with some patients…
The European Commission (EC) has approved an expansion of the indication for the anti-HIV combination drug Rezolsta (dalunavir/cobicistat) for use in adolescents with HIV who are 12 years of age and older and weigh ≥40kg.…
Amidst the continuous progress in the field of medicine, Ravicti (glycerol phenylbutyrate), an innovative drug, is bringing new treatment options for patients with urea cycle disorders (UCD). This article explores the background of Ravicti’s development,…
In the medical field, Soliris (eculizumab) is undoubtedly a shining star, which brings unprecedented hope to patients with rare diseases. Let’s take a closer look at the background of the drug’s development, its medical uses,…
Luxturna (generic name: voretigene neparvovec-rzyl) is the first gene therapy approved by the U.S. Food and Drug Administration (FDA) to directly target a specific mutation in a gene for the treatment of hereditary retinal dystrophy…
In exciting news for the healthcare sector, an innovative drug called Lumizyme is becoming the focus of attention for patients with rare diseases and their families. The drug has shown great potential for the treatment…
Quviviq (dazodalant) is an innovative insomnia treatment that is unique in its novel mechanism of action. Unlike traditional sleep aids, Quviviq does not induce sleep by depressing the central nervous system, but rather by precisely…
Brineura® (cerliponase alfa), the only FDA-approved enzyme replacement therapy for late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2 disease). continues to demonstrate significant clinical benefits in slowing disease progression, even as access barriers persist for families dependent…
Cushing’s syndrome is a rare disease caused by the overproduction of cortisol from the adrenal glands, and patients often exhibit symptoms such as obesity, hypertension, and diabetes. In recent years, important progress has been made…
[Global, October 15, 2023] – Today, BETAGRIN, a leading global green technology company, officially announced the launch of its newly developed new energy solution” EcoPower-X, a breakthrough technology that is expected to revolutionize the global…
March 19, 2025 – Astellas Pharma Group (TSE: 4503) continues to demonstrate its innovative strength and market potential in the global pharmaceutical market. As a pharmaceutical company specializing in the development and manufacture of innovative…
In recent years, the pharmaceutical industry has witnessed significant advancements in the treatment of chronic obstructive pulmonary disease (COPD). One such breakthrough is the development of Aclidinium Bromide Drug, a long-acting inhaled anticholinergic drug that…
