Amyloidosis - Biotech, Pharma and Life Science Channel
AA amyloidosis affects approximately 35,000 - 50,000 individuals in the United States, Europe (Top 5 countries) and Japan. The disease occurs in a subset of patients with long-lasting inflammatory conditions, such as rheumatic diseases (ex: rheumatoid arthritis, ankylosing spondylitis), Familial Mediterranean Fever and Crohn’s disease, in addition to certain chronic infectious diseases, such as tuberculosis and chronic bronchiectasis. Approximately 65% of AA amyloidosis patients are first diagnosed with rheumatic diseases.
Source: http://www.bellushealth.com/English/products/kiacta/default.aspx
PubMed Articles
Cramer et al. (Reports, 23 March 2012, p. 1503; published online 9 February 2012) reported that bexarotene rapidly reduces β-amyloid (Aβ) levels and plaque burden in two mouse models of Aβ depositi...
Evaluation of the mean platelet volume in secondary amyloidosis due to familial Mediterranean fever.
Familial Mediterranean fever (FMF) is an inflammatory disorder that is leading cause of secondary amyloidosis (AA). This study was designed to investigate the level of mean platelet volume (MPV) in AA...
Peripherally Applied Synthetic Peptide isoAsp7-Aβ(1-42) Triggers Cerebral β-Amyloidosis.
Intracerebral and intraperitoneal inoculation with β-amyloid-rich brain extracts originating from patients with Alzheimer's disease as well as intracerebral injection of aggregates composed of synthe...
Implantable defibrillators in cardiac amyloidosis.
Tocilizumab is a highly effective therapeutic agent for the treatment of rheumatoid arthritis and systemic juvenile idiopathic arthritis. Furthermore, a large amount of case study data reveals that to...
Senile systemic amyloidosis localized to the stomach.
We report two cases of thyroid mucosa-associated lymphoid tissue (MALT) lymphoma with associated amyloid protein deposition. While other primary thyroid neoplasms sush as medullary carcinoma and plasm...
"Idiopathic Bence-Jones proteinuria": a new characterization of an old entity.
Idiopathic Bence-Jones proteinuria (BJP) is a rare plasma cell dyscrasia, of which the clinical and biological characteristics are yet unclear. Historical data suggested that they are at higher risk o...
Pomalidomide: First Global Approval.
Pomalidomide (Pomalyst(®)) is a small molecule analogue of thalidomide under development with Celgene Corporation for the oral treatment of haematological and connective tissue diseases. Pomalidomide...
News Articles
Alnylam to Webcast Presentation at Goldman Sachs 34th Annual Global Healthcare Conference
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, today announced that management will present a company overview at the Goldman Sachs 34th...
LONDON, May 31, 2013 /PRNewswire/ -- Edison Investment Research, a leading international investment research firm, announces the initiation of full coverage of Bellus Health (TSX: BLU), a Canadian ph...
Edison Investment - Bellus Health - Initiation of coverage - Pivotal play in rare kidney disease
Bellus Health’s lead candidate, Kiacta, is in a pivotal Phase III trial for amyloid A (AA) amyloidosis, an orphan drug indication affecting up to 50,000 patients worldwide. We estimate the pro...
Prothena May Have The First Disease-Modifying Therapy For Amyloidosis
By SciFi Advisor:Amyloidoses are rare diseases characterized by the uncontrolled production of bundles of misfolded amyloid proteins, which accumulate in tissues causing organ failure and ultimately d...
-- First oral proteasome inhibitor investigated in newly diagnosed patients – Takeda Pharmaceutical Company Limited (TSE:4502) today announced the initiation of an in...
Alnylam to Webcast Presentation at Deutsche Bank 38th Annual dbAccess Health Care Conference
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, today announced that management will present a company overview at the Deutsche Bank 38th...
Alnylam to Webcast Presentation at 2013 UBS Global Healthcare Conference
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, today announced that management will present a company overview at the 2013 UBS Global He...
Betmiga, Lucentis and Elvanse accepted by SMC
Three medicines have been accepted for use in NHS Scotland by the Scottish Medicines Consortium, according to advice issued yesterday (13 May 2013). Mirabegron (Betmiga) is accepted for the sympto...
Prothena Corporation: Prothena Reports First Quarter Financial Results
Phase 1 Clinical Trial Initiated in April for NEOD001 in AL Amyloidosis Patients DUBLIN, Ireland, 2013-05-14 22:05 CEST (GLOBE NEWSWIRE) -- Prothena Corporation plc. (Nasdaq:PRTA), a clinical stage...
Alnylam to Webcast Presentation at 2013 Bank of America Merrill Lynch Health Care Conference
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, today announced that management will present a company overview at the 2013 Bank of Ameri...
Events
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Companies
Company Description: ProteoTech Inc. is a world leader in research and development of new therapeutics derived from proteoglycan and amyloid technologies for the treatment of major human diseases. Pro...
Alnylam is a biopharmaceutical company developing novel therapeutics based on RNA interference, or RNAi. The company is applying its therapeutic expertise in RNAi to address signi...
Clinical Trials
CC-5013 With or Without Dexamethasone in Treating Patients With Primary Systemic Amyloidosis
RATIONALE: Drugs such as CC-5013 and dexamethasone may be effective in treating primary systemic amyloidosis. PURPOSE: This phase II trial is studying CC-5013 to see how well it works wit...
RATIONALE: Drugs such as melphalan, thalidomide, and dexamethasone may be effective in treating patients with primary systemic amyloidosis. PURPOSE: This phase II trial is studying how we...
RATIONALE: Autologous stem cell transplantation may be effective treatment for primary systemic (AL) amyloidosis. PURPOSE: This phase II trial is studying how well tandem (two) autologous...
RATIONALE: Drugs used in chemotherapy work in different ways to stop the growth of plasma cells, either by killing the cells or by stopping them from dividing. Having a stem cell transplan...
RATIONALE: Drugs used in chemotherapy such as melphalan work in different ways to stop cancer cells from dividing so they stop growing or die. Combining chemotherapy with donor peripheral...
4'-Iodo-4'-Deoxydoxorubicin in Treating Patients With Primary Systemic Amyloidosis
RATIONALE: 4'-Iodo-4'-deoxydoxorubicin may improve organ dysfunction and ease symptoms caused by primary systemic amyloidosis. PURPOSE: Phase II trial to study the effectiveness of 4'-iod...
Dexamethasone Plus Interferon Alfa in Treating Patients With Primary Systemic Amyloidosis
RATIONALE: Chemotherapy plus interferon alfa may be effective for primary systemic amyloidosis. PURPOSE: Phase II trial to study the effectiveness of dexamethasone plus interferon alfa in...
RATIONALE: Drugs used in chemotherapy work in different ways to stop the growth of plasma cells, either by killing the cells or by stopping them from dividing. Having a peripheral stem cel...
RATIONALE: Giving bortezomib together with melphalan and dexamethasone may be an effective treatment for primary amyloidosis and light chain deposition disease. PURPOSE: This phase II tri...
RATIONALE: Giving chemotherapy, such as melphalan, before a peripheral stem cell transplant stops the growth of plasma cells by stopping them from dividing or killing them. Giving colony-s...
Videos
Amyloidosis Awareness
Originally published by CartoonMedicine here: http://vimeo.com/3358025 This short animated movie is meant to encourage better awareness and understanding of ...
Medical and Biotech [MESH] Definitions
Hereditary Autoinflammatory Diseases
Hereditary inflammation conditions, characterized by recurrent episodes of systemic inflammation. Common symptoms include recurrent fever, rash, arthritis, fatigue, and secondary AMYLOIDOSIS. Hereditary autoinflammatory diseases are associated with mutations in genes involved in regulation of normal inflammatory process and are not caused by AUTOANTIBODIES, or antigen specific T-LYMPHOCYTES.
Tdp-43 Proteinopathies
Diseases characterized by the presence of abnormally phosphorylated, ubiquitinated, and cleaved DNA-binding protein TDP-43 in affected brain and spinal cord. Inclusions of the pathologic protein in neurons and glia, without the presence of AMYLOID, is the major feature of these conditions, thus making these proteinopathies distinct from most other neurogenerative disorders in which protein misfolding leads to brain amyloidosis. Both frontotemporal lobar degeneration and AMYOTROPHIC LATERAL SCLEROSIS exhibit this common method of pathogenesis and thus they may represent two extremes of a continuous clinicopathological spectrum of one disease.
Gerstmann-straussler-scheinker Disease
An autosomal dominant familial prion disease with a wide spectrum of clinical presentations including ATAXIA, spastic paraparesis, extrapyramidal signs, and DEMENTIA. Clinical onset is in the third to sixth decade of life and the mean duration of illness prior to death is five years. Several kindreds with variable clinical and pathologic features have been described. Pathologic features include cerebral prion protein amyloidosis, and spongiform or neurofibrillary degeneration. (From Brain Pathol 1998 Jul;8(3):499-513; Brain Pathol 1995 Jan;5(1):61-75)
Cerebral Amyloid Angiopathy
A heterogeneous group of sporadic or familial disorders characterized by AMYLOID deposits in the walls of small and medium sized blood vessels of CEREBRAL CORTEX and MENINGES. Clinical features include multiple, small lobar CEREBRAL HEMORRHAGE; cerebral ischemia (BRAIN ISCHEMIA); and CEREBRAL INFARCTION. Cerebral amyloid angiopathy is unrelated to generalized AMYLOIDOSIS. Amyloidogenic peptides in this condition are nearly always the same ones found in ALZHEIMER DISEASE. (from Kumar: Robbins and Cotran: Pathologic Basis of Disease, 7th ed., 2005)
Technetium Tc 99m Pyrophosphate
A radionuclide imaging agent used primarily in scintigraphy or tomography of the heart to evaluate the extent of the necrotic myocardial process. It has also been used in noninvasive tests for the distribution of organ involvement in different types of amyloidosis and for the evaluation of muscle necrosis in the extremities.
