Recently, Qelbree has attracted a lot of attention in the pharmaceutical field.Qelbree focuses on specific attention-related conditions. The development of this drug has been a long and rigorous process, with researchers investing a great deal…
On 15 November 2024, the US Food and Drug Administration (FDA) approved a new drug called Revuforj (revumenib) for the treatment of adult and paediatric patients aged 1 year and older with relapsed or refractory…
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.…
FDA Approval Of Rylaze Fills Asparaginase Shortage Gap, Offers New Hope For ALL/LBL Patients In June 2021, the U.S. Food and Drug Administration (FDA) approved Rylaze (JZP458, recombinant Owenella asparaginase) for the treatment of patients…
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,…
Myalepta (generic: metreleptin), the first approved leptin replacement therapy, is offering new hope for these patients. Lipodystrophy is a rare metabolic disorder in which patients suffer from severe metabolic disturbances due to leptin deficiency. Global…
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…
Folotyn (generic name: Pralatrexate), the first drug approved by the U.S. Food and Drug Administration (FDA) for the treatment of relapsed or refractory peripheral T-cell lymphoma (PTCL), has made a number of important advances worldwide…
