March 07, 2025 – On January 30, 2025, the U.S. Food and Drug Administration (FDA) approved Journavx (Suzetrigine), 50 milligram oral tablets, a first-in-class non-opioid analgesic, to treat moderate to severe acute pain in adults. Journavx (Suzetrigine) reduces pain by targeting a …
Journavx (Suzetrigine): Non-Opioid Pain Treatment Appoved by FDA Read more »
November 25, 2023 – Sickle cell anemia is an inherited disease of red blood cells (erythrocytes). It belongs to the group of hemoglobinopathies (hemoglobin disorders) and leads to corpuscular hemolytic anemia. Affected people have a mutation in the hemoglobin β …
Sickle cell anemia: Genetic scissors drug approved for first time Read more »
April 12, 2020 – The human genetic disease neurofibromatosis type 1 (NF-1) is a complex multi-system disorder caused by the mutation of a gene (NF1 gene) on chromosome 17 that is responsible for production of a protein called neurofibromin which is …
First therapy for children with debilitating NF-1 approved in the USA Read more »
May 30, 2019 – The American Food and Drug Administration (FDA) recently approved Alpelisib (Piqray) to be used in combination with the FDA-approved endocrine therapy fulvestrant, to treat postmenopausal women, and men, with hormone receptor (HR)-positive, human epidermal growth factor receptor …
The PI3K inhibitor Alpelisib (Piqray) for progressing breast cancer in women (and men) Read more »
May 29, 2019 – Spinal muscular atrophy (SMA) is a group of neuromuscular disorders that result in the loss of motor neurons and progressive muscle wasting. The severity of symptoms and age of onset varies by the type. Some types are apparent at …
Gene therapy: Zolgensma to treat spinal muscular atrophy Read more »
March 31, 2019 – The American Food and Drug Administration (FDA) has just approved drugs for two rather problematic neurological conditions, namely multiple sclerosis (i.e., Cladribine (Mavenclad)) and non-radiographic axial spondyloarthritis (i.e., Certolizumab pegol (Cimzia)), offering patients with these conditions …
New treatments for multiple sclerosis and inflammatory arthritis approved Read more »
February 07, 2019 – The American Food and Drug Administration (FDA) just approved Caplacizumab-yhdp (Cablivi), the first therapy specifically indicated, in combination with plasma exchange and immunosuppressive therapy, for the treatment of adult patients with acquired thrombotic thrombocytopenic purpura (aTTP), …
Caplacizumab-yhdp (Cablivi): the first therapy for the treatment of aTTP Read more »
January 12, 2018 – Fabry disease is an inherited disorder caused by mutations (alterations) in the alpha-galactosidase A (GLA) gene located on the X-chromosome. Fabry disease is rare and affects both males and females. It is estimated that classic Fabry disease …
Migalastat (Galafold): New treatment for Fabry disease Read more »
December 30, 2018 – Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is an aggressive and rare disease of the bone marrow and blood that can affect multiple organs, including the lymph nodes and the skin and often presents as or evolves …
December 04, 2018 – The American Food and Drug Administration (FDA) has just approved its first ever treatment, Amifampridine (Firdapse), for Lambert-Eaton myasthenic syndrome (LEMS), a rare autoimmune disorder for adult patients. LEMS affects the connection between nerves and muscles …
First ever treatment for Lambert-Eaton myasthenic syndrome (LEMS) FDA approved Read more »