Diazoxide, sold under brand names such as Proglycem, is a medication used to treat hypoglycemia caused by a number of specific reasons. This includes unresectable islet cell tumors and leucine sensitivity. It can also be…
Covalizumab, sold under the brand name Piasky, is a monoclonal antibody used to treat paroxysmal sleep hemoglobinuria. Piasky Crovalimab – A New Hope For The Treatment of Blood Disorders An exciting news comes in the…
Nitisinone capsules are a hydroxyphenylpyruvate dioxygenase inhibitor combined with tyrosine and phenylalanine dietary restrictions for the treatment of tyrosinemia type I (HT-1) in adults and children. Ureolaric Acid Oxidase Deficiency: A Rare Genetic Disorder HT-1…
Sirolimus Gel is used for the treatment of tuberous sclerosis-associated facial angiofibromas in pediatric and adult patients 6 years of age and older. With the mechanism of action of sirolimus and remarkable clinical efficacy, this…
Spesolimab Injection (trade name: Spevigo), in a subcutaneous injection formulation, for the reduction of exacerbations of generalized pustular psoriasis (GPP) in adolescents (weighing ≥40kg) and adults 12 years of age and older. Boehringer Ingelheim announced…
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…
As the first biologic agent approved for complement system regulation, eculizumab injection has made a milestone breakthrough in the treatment of refractory diseases such as Paroxysmal Nocturnal Hemoglobinuria (PNH) and Atypical Hemolytic Uremic Syndrome (aHUS),…
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…
