Miglustat Gen.Orph is indicated for the oral treatment of adult patients with mild to moderate type 1 Gaucher disease. Miglustat Gen.Orph may be used only in the treatment of patients for whom enzyme replacement therapy is unsuitable (see sections 4.4 and 5.1). Miglustat Gen.Orph is indicated for the treatment of progressive neurological manifestations in adult patients and paediatric patients with Niemann-Pick type C disease (see sections 4.4, and 5.1).
The FDA has cleared the Panther system from Gen-Probe. The Panther is a fully automated and integrated molecular testing platform...
Medtronic’s newest generation self-expanding, transcatheter aortic valve replacement (TAVR) system, Evolut FX, significantly improved commissure alignment during TAVR procedures compared to earlier generation Evolut systems. A late breaker presentation at PCR London Valves 2022 summarized the early limited market release experience with the Evolut FX system in 168 patients from six US centers
Ionis Pharmaceuticals announced its partner, Roche, has decided to discontinue dosing in the Phase III GENERATION HD1 study of IONIS-HTTRx (tominersen) in manifest Huntington's disease (HD).
Sol-Gel Technologies, Ltd. announced that the FDA has approved its first proprietary drug product, Twyneo (tretinoin/benzoyl peroxide) cream, 0.1%/3%, indicated for the treatment of acne vulgaris in adults and pediatric patients nine years of age and older.
Vertex Pharmaceuticals Incorporated announced that the European Commission has granted approval of the label extension for Kaftrio (ivacaftor/tezacaftor/elexacaftor) in a combination regimen with ivacaftor for the treatment of cystic fibrosis (CF) in all patients ages 12 years and older who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
A new study has found individuals addicted to prescription Opioid painkillers are more likely to succeed in treatment with the...
Irritable bowel syndrome (IBS) is a complex functional gastrointestinal disorder that is exceedingly common in clinical practice. IBS with predominant constipation (IBS-C) is a subtype of IBS that accounts for more than a third of the IBS diagnosed.
Melinta Therapeutics, LLC announced that the FDA has approved Kimyrsa (oritavancin) for the treatment of adult patients with acute bacterial skin and skin structure infections (ABSSSI) caused by susceptible isolates of designated Gram-positive microorganisms, including methicillin-resistant Staphylococcus aureus (MRSA).