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To treat Alzheimer's disease [1] Loqtorzi: To treat recurrent or metastatic nasopharyngeal carcinoma with or following other therapies [1] Ogsiveo: To treat desmoid tumors [1] Pombiliti: To treat late-onset Pompe disease with miglustat [1] Rivfloza: To lower urinary oxalate levels in primary hyperoxaluria type 1 and relatively preserved kidney ...
Exagamglogene autotemcel (Casgevy): treatment for sickle cell disease. [11] Gendicine: treatment for head and neck squamous cell carcinoma; Idecabtagene vicleucel (Abecma): treatment for multiple myeloma [12] Lovotibeglogene autotemcel (Lyfgenia): treatment for sickle cell disease. [11] Nadofaragene firadenovec (Adstiladrin): treatment for ...
(Reuters) -The U.S. Food and Drug Administration has approved Ionis Pharmaceuticals' drug to treat a rare genetic disorder, making it the company's first wholly-owned drug, the health regulator's ...
Given these parameters, exon skipping can be used to restore an open reading frame by inducing a deletion of one or several exons within the central rod domain, and thus converting a DMD phenotype into a BMD phenotype. The genetic mutation that leads to Becker muscular dystrophy is an in-frame deletion. This means that, out of the 79 exons that ...
The initial list includes common prescriptions such as penicillin, metformin, lithium and albuterol asthma inhalers, as well as drugs for high cholesterol, high blood pressure and other chronic ...
Since at least 1974, the deletion in the long arm of chromosome 5 has been known to be associated with dysplastic abnormalities of hematopoietic stem cells. [24] [25] By 2005, lenalidomide, a chemotherapy drug, was recognized to be effective in MDS patients with the 5q- syndrome, [26] and in December 2005, the US FDA approved the drug for this ...
The problem with rare diseases. A genetic mutation disease like HNRNPH2, estimated at about 150 cases globally, lacks enough patients to make finding a cure profitable for pharmaceutical companies ...
The first medication approved to treat seizures associated with CDD is ganaxolone (Ztalmy), approved by the FDA in March 2022 [25] and by the EMA in July 2023. [26] Other efforts to develop small molecule therapeutics for CDD include phase 2 and phase 3 trials already underway or completed, and others in earlier stages of development.