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Medication
Tofersen, sold under the brand name Qalsody, is a medication used for the treatment of amyotrophic lateral sclerosis (ALS). Tofersen is an antisense oligonucleotide
Tofersen
Rare neurodegenerative disease
familial ALS, especially due to the availability of genetic medicine (tofersen) for carriers of SOD1 ALS. A shortage of genetic counselors and limited
ALS
Biotechnology company
and Drug Administration (FDA) granted accelerated approval to Qalsody (tofersen) for the treatment of amyotrophic lateral sclerosis (ALS) in adults with
Ionis_Pharmaceuticals
American pharmaceutical company
(Skyclarys) for the treatment of Friedreich's ataxia (4.0% of 2024 revenues); tofersen (Qalsody) for the treatment of ALS; and dimethyl fumarate (Fumaderm) for
Biogen
Form of treatment for genetic disorders and other illnesses
greatly enhances delivery efficiency and cell-type specific targeting. Tofersen (marketed as Qalsody) was approved by the FDA for the treatment of SOD1-
Antisense_therapy
Pharmaceutical drug classification
Sodium phenylbutyrate and ursodoxicoltaurine N07XX21 Eplontersen N07XX22 Tofersen N07XX23 Troriluzole N07XX24 Trofinetide N07XX25 Omaveloxolone N07XX26 Pridopidine
ATC_code_N07
Study of role of genetics in the nervous system
(AAV) vectors. Similarly, antisense oligonucleotide (ASO) therapies like tofersen and nusinersen work by modulating RNA splicing or reducing toxic protein
Neurogenetics
IONIS-GCCRRx, nusinersen, oblimersen, olezarsen, pelacarsen, sefaxersen, tofersen, and volanesorsen. First-generation anti-miRNA oligonucleotides utilized
Phosphorothioate_nucleic_acid
TOFERSEN
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Girl/Female
Australian, French, Portuguese
Invaluable
Girl/Female
Muslim/Islamic
Light of the moon
Girl/Female
Bengali, Indian
Goddess
Boy/Male
Indian, Punjabi, Sikh
Earning the Wealth of Naam
Female
Hawaiian
Hawaiian name KEIKI means "child."
Girl/Female
Hindu
Goddess Durga
Boy/Male
Tamil
Parijata | பாரீஜாத
Tarumoolastha dweller under the Parijata tree
Girl/Female
Australian, Danish, Finnish, German, Hebrew, Swedish
Noble; Warfare; Battle; Female Warrior
Female
English
Short form of English Cheryl, probably CHER means "darling beryl."
Boy/Male
Muslim
Inimitability of the truth
TOFERSEN
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