Enspryng Satralizumab Injection is indicated for the treatment of neuromyelitis optica spectrum disease (NMOSD) in adolescents ≥12 years of age and adult patients with antibody-positive aquaporin 4 (AQP4). Enspryng Satralizumab to Challenge Rare Diseases The…
Ozamode hydrochloride capsules (Ozanimode) are used for the treatment of adults with relapsing forms of multiple sclerosis, including clinically isolated syndrome, relapsing-remitting multiple sclerosis, and active secondary progressive multiple sclerosis. Technological Innovation – Precision Medicine…
The outstanding achievement of siltuximab for injection in the treatment of Castleman’s disease not only brings new hope for treating this rare disease but also marks an important step forward in global medical science and…
Alglucosidase alfa, the brand name Myozyme, is currently the only injectable and potent drug for the rare disease Pompe in the world. Alglucosidase alfa was developed by Chen Yuan-Chung, Director of the Institute of Biomedical…
Risdiplam Oral Solution in Bulk (Emancine) is indicated for the treatment of spinal muscular atrophy (SMA) in patients 2 months of age and older. With rapid advances in biotechnology, the field of rare diseases has…
On June 30, 2023, China’s National Drug Administration (NMPA) formally approved Pitolisant hydrochloride tablets (Sharkos®) for the treatment of excessive daytime sleepiness (EDS) or sudden collapse in adult patients with episodic sleepiness (Narcolepsy). Tiloxan has…
Nusinersen Injection (Nusinersen): a beacon of hope for spinal muscular atrophy patients, marketed under the name Spinraza, is a medication for the treatment of spinal muscular atrophy (SMA), a rare neuromuscular disorder. In December 2016,…
Fabry disease is a rare, life-threatening genetic disorder that affects individuals across all age groups. Characterized by a deficiency in the enzyme α-galactosidase A, this condition results in the accumulation of harmful substances in the…
