FDA approves first-of-its kind targeted RNA-based therapy to treat a rare disease

FDA approves new drug for treatment of polyneuropathy caused by hereditary transthyretin-mediated amyloidosis (hATTR). This is the first FDA-approved treatment for this rare, debilitating and often fatal genetic disease and the first FDA approval of a new class of drugs called small interfering ribonucleic acid (siRNA) treatment.
Source: Food and Drug Administration - Category: American Health Source Type: news

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Authors: Rizk M, Tüzmen S Abstract Onpattro, also commonly known as patisiran, is a small interfering RNA (siRNA) molecule packaged within a lipid nanoparticle and is transported into the cell to target transthyretin gene (TTR) messenger mRNA (mRNA) by attaching to its complementary sequence. The target mRNA is degraded and both mutant and wild-type amyloid transthyretin (ATTR) protein production becomes suppressed. This drug was developed by Alnylam Pharmaceuticals to treat a rare disease called hereditary ATTR (hATTR) amyloidosis. This disease develops as a result of the deposition of toxic aggregates of mis...
Source: Drugs of Today - Category: Drugs & Pharmacology Tags: Drugs Today (Barc) Source Type: research
ConclusionAmyloid PET tracers such as18F-flutemetamol could be a promising tool in diagnosing and in therapy response assessment for patients with cardiac amyloidosis.
Source: Annals of Nuclear Medicine - Category: Nuclear Medicine Source Type: research
Hereditary transthyretin amyloidosis (ATTRv amyloidosis) is caused by a variant transthyretin (TTR), which is a serum protein secreted by the liver. Mass spectrometry (MS) is a useful tool that can detect vari...
Source: Orphanet Journal of Rare Diseases - Category: Internal Medicine Authors: Tags: Research Source Type: research
MONDAY, May 6, 2019 -- Vyndaqel (tafamidis meglumine) and Vyndamax (tafamidis) capsules have been approved to treat adults with cardiomyopathy caused by transthyretin mediated amyloidosis (ATTR-CM), the U.S. Food and Drug Administration announced...
Source: Drugs.com - Pharma News - Category: Pharmaceuticals Source Type: news
On May 3, the U.S. Food and Drug Administration approved Vyndaqel (tafamidis meglumine) and Vyndamax (tafamidis) capsules for the treatment of the heart disease (cardiomyopathy) caused by transthyretin mediated amyloidosis (ATTR-CM) in adults. These are the first FDA-approved treatments for ATTR-CM. Vyndaqel and Vyndamax have the same active moiety, tafamidis, but they are not substitutable on a milligram to milligram basis and their recommended doses differ.
Source: World Pharma News - Category: Pharmaceuticals Tags: Featured FDA Regulatory Affairs Source Type: news
Conclusions: We have shown that severity of MWS symptoms gradually increased with age toward distinct generation-specific phenotypes. A uniform trajectory of disease evolution has encouraged us to postpone institution of IL-1 blockade in affected oligosymptomatic children. This report illustrates importance of close interdisciplinary collaboration. Introduction Cryopyrinopathies (Cryopyrin Associated Periodic Syndromes, CAPS) belong to autoinflammatory disorders with autosomal dominant inheritance caused by the gain-of-function point mutation of NLRP3 (NACHT, LRR, and PYD domains-containing protein 3) gene which en...
Source: Frontiers in Immunology - Category: Allergy & Immunology Source Type: research
Bill Crumplin, with photo of his late wife Donna Williams, image courtesy Sudbury.comYou might recall mypost of a few years ago about Sudbury, Ontario, Canada, a medium-sized town that turned to its citizens to fund a PET/CT facility when the much-touted Health Service would not provide it. The wait-time was not just an inconvenience; the health of Sudbury citizens was adversely affected by the lack of local scanning capability.The wait goes on, it seems.My friend Stacey discoveredanother such tragedy related to imaging, or rather, lack thereof. As reported onSudbury.com:Donna Williams ’ dying wish was to raise money...
Source: Dalai's PACS Blog - Category: Radiology Source Type: blogs
In honor of Rare Disease Day, Alnylam Pharmaceuticals has launched a grant program for patients advocacy groups increasing awareness of two uncommon diseases. Alnylam (Nasdaq: ALNY) announced the first seven recipients of its new Advocacy for Impact Grants program Thursday. Through the program, the drug company will give out $248,000 to organizations focused on the rare diseases acute hepatic porphyria and ATTR amyloidosis. The activities of those grou ps include advocating for better diagnosis…
Source: bizjournals.com Health Care:Pharmaceuticals headlines - Category: Pharmaceuticals Authors: Source Type: news
Conclusions: Adult periocular colloid milium is an unusual presentation of a rare cutaneous deposition disease. The occurrence of conjunctival colloid milium is rare, and we report this case to increase knowledge and management regarding this rare disease and to differentiate it from other diseases such as amyloidosis, lipoid proteinosis, plasmacytoma, and conjunctival lymphoma, which carry a graver prognosis, and have a similar clinical appearance and can be distinguished only on the basis of histopathological characteristics.
Source: Cornea - Category: Opthalmology Tags: Case Report Source Type: research
IgM related AL amyloidosis is a rare disease with patients presenting with more renal and neurological involvement and less cardiac involvement compared to those with non-IgM related disease. We retrospectively reviewed 38 patients receiving autologous stem cell transplant (ASCT) for IgM related AL amyloidosis at the Mayo Clinic between May 1999 and June 2018. Median age was 61 years and 71% were male. The most common organs involved were renal (63%), neurological (32%) and cardiac (26%). The median difference between involved and uninvolved free light chains (dFLC) was 6.2 mg/dL and most patients had early mayo stage (87%...
Source: Biology of Blood and Marrow Transplantation - Category: Hematology Authors: Tags: 588 Source Type: research
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