Quick Facts about Netherton Syndrome Netherton syndrome, a less common form of ichthyosis, is a monogenic cutaneous condition characterized by congenital scaly erythroderma, evolving into typical e...
Orchard Therapeutics’ Biologics License Application for OTL-200, a gene therapy under investigation for metachromatic leukodystrophy treatment, has been accepted by the FDA. This rare disease treatment received approval in Europe back in 2020. Notably, the FDA has granted Orchard’s application Priority Review statu...
Find MoreHuntington’s disease is an incurable, rare genetic, progressive neurodegenerative disorder. According to the National Organization for Rare Disorders (NORD), about 30,000 people in the United States have Huntington’s disease, and another 200,000 are at risk of developing the condition. As per DelveInsight analy...
Find MoreFDA Grants Breakthrough Therapy Designations to Trastuzumab Deruxtecan for HER2+ Solid Tumors, Including mCRC ENHERTU® (fam-trastuzumab deruxtecan-nxki) has been granted two additional Breakthrough Therapy Designations (BTDs) in the United States for the treatment of adult patients with unresectable or metastati...
Find MoreFabry disease is a rare hereditary lysosomal storage disorder that is caused by mutation in the GLA gene located on the X chromosome. The defect leads to the deficiency of an enzyme called alpha-galactosidase A, responsible for the breakdown of globotriaosylceramide (Gb3). The accumulation of the fatty substance le...
Find MoreHomozygous familial hypercholesterolemia (HoFH) is a rare form of familial hypercholesterolemia, an autosomal-dominant genetic disorder of lipid metabolism characterized by strikingly elevated levels of low-density lipoprotein cholesterol (LDL-C). Heterozygous Familial Hypercholesterolemia vs. Homozygous F...
Find MoreEylea HD Injection 8 Mg Approved By FDA for Treatment of Wet AMD, DME, and Diabetic Retinopathy The FDA has approved Regeneron Pharmaceuticals’ EYLEA HD (aflibercept) Injection of 8 mg for the treatment of patients with wet age-related macular degeneration (wAMD), diabetic macular edema (DME), and diabetic retin...
Find MoreFDA Clears J&J’s 2-in-1 Tablet for Prostate Cancer Johnson & Johnson's Janssen Pharmaceutical Companies stated that the US Food and Drug Administration (FDA) had approved AKEEGA (niraparib and abiraterone acetate), the first-and-only dual-action tablet combining a PARP inhibitor including abiraterone ace...
Find MoreIn recent years, significant strides have been made in the realm of medical imaging, ushering in a new era of possibilities for healthcare professionals engaged in diagnosing, treating, and monitoring a diverse array of medical conditions and diseases. The convergence and developments in the state-of-the-art techno...
Find MoreFDA Expands GSK’s Jemperli Approval for Endometrial Cancer GSK plc announced that the US Food and Drug Administration (FDA) has approved Jemperli (dostarlimab) in combination with carboplatin and paclitaxel, followed by Jemperli as a single agent for the treatment of adult patients with mismatch repair deficient...
Find MoreAnorexia Nervosa (AN) is a psychological condition that is characterized by an abnormally low weight.....
Find MoreThe medical device industry is a dynamic, innovative, and heterogeneous sector. The MedTech industry.....
Find MoreA degenerative disease, Exocrine Pancreatic Insufficiency (EPI), is a consequence of insufficient pr.....
Find MoreRNAi is a fundamental gene-silencing pathway in eukaryotic cells, where long pieces of double-strand.....
Find MoreVascular Dementia (VaD) is a common type of Dementia caused by reduced blood flow to the brain. VaD .....
Find MoreSystemic Lupus Erythematosus (SLE) is an autoimmune disease where the immune system of the body mist.....
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