Patisiran, sold under the brand name Onpattro, is a medication used for the treatment of polyneuropathy in people with hereditary transthyretin-mediated...
15 KB (952 words) - 06:33, 10 March 2024
Alnylam Pharmaceuticals (section FDA on patisiran)
In late 2016, the company's lead candidate in phase III studies was patisiran, a treatment targeting transthyretin (TTR) for the treatment of TTR-mediated...
20 KB (1,743 words) - 17:48, 29 May 2024
European Union and in the USA. Patisiran functions similarly to inotersen. Moderate-certainty evidence suggests that patisiran mitigates worsening of peripheral...
55 KB (5,426 words) - 23:43, 23 June 2024
vutrisiran to patisiran. 164 ATTRv amyloidosis patients were randomized 3:1 to subcutaneous vutrisiran 25 mg every 3 months or patisiran 0.3 mg/kg IV infusion...
26 KB (2,599 words) - 16:32, 16 July 2024
disorders. Patisiran is the first double stranded siRNA-based medication approved in 2018 and developed by Alnylam Pharmaceuticals. Patisiran uses the RNAi...
141 KB (15,474 words) - 08:09, 11 August 2024
and medications that decreases the activity of the transthyretin genes (patisiran and inotersen). In patients with familial transthyretin mutations, liver...
30 KB (3,354 words) - 02:30, 21 June 2024
phospholipase 2G6-associated neurodegeneration (PLAN). The FDA granted Patisiran (Onpattro) orphan drug status and breakthrough therapy designation due...
35 KB (4,197 words) - 13:34, 26 September 2023
first company to have a siRNA therapy approved by the FDA. Onpattro (patisiran) was approved for the treatment of polyneuropathy of hereditary transthyretin-mediated...
56 KB (6,826 words) - 08:01, 15 August 2024
Fampridine N07XX08 Tafamidis N07XX10 Laquinimod N07XX11 Pitolisant N07XX12 Patisiran N07XX13 Valbenazine N07XX14 Edaravone N07XX15 Inotersen N07XX16 Deutetrabenazine...
2 KB (282 words) - 23:28, 21 December 2023
developed by Ionis Pharmaceuticals and licensed to Akcea Therapeutics. Patisiran (sold under Onpattro) was developed by Alnylam Pharmaceuticals, and also...
23 KB (2,219 words) - 02:24, 20 August 2024
number NCT01960348 for "APOLLO: The Study of an Investigational Drug, Patisiran (ALN-TTR02), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis"...
20 KB (2,382 words) - 16:13, 22 January 2024
transthyretin familial amyloid polyneuropathy. In August 2018, the FDA approved patisiran, an siRNA-based treatment, at an expected cost of up to $450,000 per year...
14 KB (1,486 words) - 16:53, 7 February 2024
siRNA drugs. In 2018, the FDA approved Alnylam's siRNA drug Onpattro (patisiran), the first drug to use LNPs as the drug delivery system. By that point...
23 KB (2,629 words) - 15:37, 19 March 2024
million in Alnylam. Under the deal, Genzyme obtained further rights to patisiran, an RNAi treatment for transthyretin-mediated amyloidosis – a condition...
91 KB (8,778 words) - 06:05, 10 August 2024
many gene therapy based drugs became available, such as Zolgensma and Patisiran. Most of these approaches utilize viral vectors, such as adeno-associated...
95 KB (10,491 words) - 00:01, 9 August 2024
Mogamulizumab-kpkc Kyowa Hakko Kirin mycosis fungoides or Sézary syndrome Patisiran Alnylam Pharmaceuticals hereditary transthyretin-mediated amyloidosis...
34 KB (180 words) - 22:09, 8 April 2024
agreement with lower milestones and royalties, which covers Alnylam's product patisiran. Tekmira caught the world's attention and its stock rose dramatically...
12 KB (1,112 words) - 17:25, 19 February 2024
how to improve the efficiency of the delivery of naked mRNA in vivo. patisiran givosiran lumasiran inclisiran Antisense RNA is the non-coding and single-stranded...
77 KB (8,886 words) - 09:03, 10 June 2024
therapies to market and developed the first-ever FDA approved RNAi drug, Patisiran, gaining FDA approval in August 2018. In 2014, Dr. Zamore co-founded another...
14 KB (1,167 words) - 18:07, 29 April 2024
compensate for the RPE65 mutation (US approval granted 2017). siRNA medicines: Patisiran (Onpattro) for the treatment of polyneuropathy in people with hereditary...
78 KB (8,788 words) - 19:06, 6 August 2024
years, but it wasn’t until 2018 that the EMA approved the drug called Patisiran/Onpattro. The drug uses ds-siRNA as a mechanism of action and is deemed...
42 KB (5,353 words) - 18:06, 29 April 2024