Viltolarsen, sold under the brand name Viltepso, is a medication used for the treatment of Duchenne muscular dystrophy (DMD). Viltolarsen is a Morpholino antisense oligonucleotide.The most common side effects include upper respiratory tract infection, injection site reaction, cough, and pyrexia (fever).Viltolarsen was approved for medical use in the United States in August 2020. After golodirsen was approved in December 2019, viltolarsen is the second approved targeted treatment for people with this type of mutation in the United States. Approximately 8% of people with DMD have a mutation that is amenable to exon 53 skipping. Medical uses
Viltolarsen is indicated for the treatment of Duchenne muscular dystrophy (DMD) in people who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping.DMD is a rare genetic disorder characterized by progressive muscle deterioration and weakness. It is the most common type of muscular dystrophy. DMD is caused by mutations in the DMD gene that results in an absence of dystrophin, a protein that helps keep muscle cells intact. The first symptoms are usually seen between three and five years of age and worsen over time. DMD occurs in approximately one out of every 3,600 male infants worldwide; in rare cases, it can affect females. Adverse effects
The most common side effects include upper respiratory tract infection, injection site reaction, cough, and pyrexia (fever).Although kidney toxicity was not observed in the clinical studies, the clinical experience is limited, and kidney toxicity, including potentially fatal glomerulonephritis, has been observed after administration of some antisense oligonucleotides.
Enophthalmos is a posterior displacement of the eyeball within the orbit. It is due to either enlargement of the bony orbit and/or reduction of the orbital content, this in relation to each other.It should not be confused with its opposite, exophthalmos, which is the anterior displacement of the eye. It may be a congenital anomaly, or be acquired as a result of trauma (such as in a blowout fracture of the orbit), Horners syndrome (apparent enophthalmos due to ptosis), Marfan syndrome, Duanes syndrome, silent sinus syndrome or phthisis bulbi. References
Further reading
Cline RA, Rootman J (1984). "Enophthalmos: a clinical review". Ophthalmology. 91 (3): 229–37. doi:10.1016/s0161-6420(84)34299-3. PMID 6717910. == External links ==