anti-cancer drug safe and effective for treating light chain (AL) amyloidosis
(Boston University School of Medicine) There's a new treatment option available for patients with AL amyloidosis: daratumumab. Studied in a prospective clinical trial, only one of the two clinical trials of this agent in AL amyloidosis worldwide, researchers have found this anti-cancer drug to be well tolerated and effective in patients with relapsed AL amyloidosis when used with appropriate pre- and post-infusion medications. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - January 27, 2020 Category: International Medicine & Public Health Source Type: news

ASNC, ASE win CMS recognition for ImageGuide Registry
The American Society of Nuclear Cardiology (ASNC) and the American Society...Read more on AuntMinnie.comRelated Reading: ASNC releases directive on cardiac amyloidosis imaging Imaging groups oppose cuts in myocardial PET rates ACC, ASE release echocardiography training guidance ASNC names Beanlands as president (Source: AuntMinnie.com Headlines)
Source: AuntMinnie.com Headlines - January 22, 2020 Category: Radiology Source Type: news

Ionis Appoints Onaiza Cadoret-Manier As Chief Corporate Development and Commercial Officer
CARLSBAD, Calif., Jan. 9, 2020 -- (Healthcare Sales & Marketing Network) -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS), the leader in RNA-targeted therapeutics, today announced the appointment of Onaiza Cadoret-Manier as chief corporate development and co... Biopharmaceuticals, Neurology, Personnel Ionis Pharmaceuticals, RNA-targeted, antisense, transthyretin amyloidosis (Source: HSMN NewsFeed)
Source: HSMN NewsFeed - January 9, 2020 Category: Pharmaceuticals Source Type: news

Treatment of Cardiac Transthyretin Amyloidosis: An Update Treatment of Cardiac Transthyretin Amyloidosis: An Update
This article presents an overview of several new pharmacological agents available to treat cardiac transthyretin amyloidosis, a rare and difficult to diagnose genetic disorder.European Heart Journal (Source: Medscape Today Headlines)
Source: Medscape Today Headlines - January 1, 2020 Category: Consumer Health News Tags: Cardiology Journal Article Source Type: news

Subtle Cognitive Difficulties May Predict Amyloidosis
TUESDAY, Dec. 31, 2019 -- Individuals with objectively-defined subtle cognitive difficulties (Obj-SCD) have faster amyloid accumulation and faster entorhinal cortical thinning compared with cognitively normal (CN) individuals, according to a study... (Source: Drugs.com - Pharma News)
Source: Drugs.com - Pharma News - December 31, 2019 Category: Pharmaceuticals Source Type: news

C2N Diagnostics Brings Home Positive Results for Alzheimer ’s Test
C2N Diagnostics’s detection test for Alzheimer’s disease is showing off some strong data coming out of the 12th Annual Clinical Trials in Alzheimer’s Disease conference. The St. Louis-based company said results from a study show the Aptus-Aβ test strongly predicts the presence of brain amyloidosis in a diverse population. C2N Diagnostics said its blood-based in vitro diagnostic is being developed to predict amyloid PET scan results. It combines into a ratio, the concentration of amyloid beta (Aβ) isoforms Aβ42 and Aβ40 in ...
Source: MDDI - December 10, 2019 Category: Medical Devices Authors: Omar Ford Tags: IVD Source Type: news

Medical News Today: What is amyloidosis?
Amyloidosis is a condition that causes abnormal proteins to grow on a person's organs. It can cause a variety of symptoms, depending on the organs involved. Learn more here. (Source: Health News from Medical News Today)
Source: Health News from Medical News Today - October 24, 2019 Category: Consumer Health News Tags: Endocrinology Source Type: news

Changes in the Blood Could Hold Key for Better Alzheimer ’s Detection
Researchers at the University of Turku have discovered new changes in blood samples associated with Alzheimer’s disease. A new study was conducted on disease-discordant Finnish twin pairs: one sibling suffering from Alzheimer's disease and the other being cognitively healthy. The researchers used the latest genome-wide methods to examine the twins' blood samples for any disease-related differences in epigenetic marks which are sensitive to changes in environmental factors. These differences between the siblings were discovered in multiple different genomic regions. Development of the late-onset form of Al...
Source: MDDI - October 15, 2019 Category: Medical Devices Authors: MDDI Staff Tags: R & D Source Type: news

AL Amyloidosis: Advances in Diagnostics and Treatment AL Amyloidosis: Advances in Diagnostics and Treatment
Recent advances have led to promising new diagnostic strategies and treatment options for AL amyloidosis.Nephrology Dialysis Transplantation (Source: Medscape Today Headlines)
Source: Medscape Today Headlines - October 2, 2019 Category: Consumer Health News Tags: Nephrology Journal Article Source Type: news

Cardiac amyloidosis — Treatment options
(Source: MayoClinic.com Full Feed)
Source: MayoClinic.com Full Feed - September 27, 2019 Category: Consumer Health News Source Type: news

Cardiac amyloidosis — What is amyloid and how does it affect the heart
(Source: MayoClinic.com Full Feed)
Source: MayoClinic.com Full Feed - September 27, 2019 Category: Consumer Health News Source Type: news

ASNC releases directive on cardiac amyloidosis imaging
The American Society of Nuclear Cardiology (ASNC) has published a set of recommendations...Read more on AuntMinnie.comRelated Reading: Imaging groups oppose cuts in myocardial PET rates ASNC names Beanlands as president ASNC releases new guidelines for SPECT MPI SNMMI, ASNC offer guidelines for myocardial perfusion imaging with PET ASNC, SNMMI advise on cardiac sarcoidosis (Source: AuntMinnie.com Headlines)
Source: AuntMinnie.com Headlines - September 9, 2019 Category: Radiology Source Type: news

Transthyretin Cardiac Amyloidosis: The Truth Is Unfolding Transthyretin Cardiac Amyloidosis: The Truth Is Unfolding
New technologies have determined that transthyretin cardiac amyloidosis, once thought to be rare and untreatable, is more common than previously thought, and novel therapeutic options are emerging.Circulation (Source: Medscape Today Headlines)
Source: Medscape Today Headlines - August 26, 2019 Category: Consumer Health News Tags: Cardiology Journal Article Source Type: news

A vicious cycle of {beta} amyloid-dependent neuronal hyperactivation
β-amyloid (Aβ)–dependent neuronal hyperactivity is believed to contribute to the circuit dysfunction that characterizes the early stages of Alzheimer’s disease (AD). Although experimental evidence in support of this hypothesis continues to accrue, the underlying pathological mechanisms are not well understood. In this experiment, we used mouse models of Aβ-amyloidosis to show that hyperactivation is initiated by the suppression of glutamate reuptake. Hyperactivity occurred in neurons with preexisting baseline activity, whereas inactive neurons were generally resistant to Aβ-mediated hyperact...
Source: ScienceNOW - August 8, 2019 Category: Science Authors: Zott, B., Simon, M. M., Hong, W., Unger, F., Chen-Engerer, H.-J., Frosch, M. P., Sakmann, B., Walsh, D. M., Konnerth, A. Tags: Medicine, Diseases, Neuroscience r-articles Source Type: news

New Alzheimer's Blood Test 94% Accurate New Alzheimer's Blood Test 94% Accurate
When combined with age and APOE ε 4 status, the blood test is 94% accurate in detecting brain amyloidosis and may be a"game changer" for the field.Medscape Medical News (Source: Medscape Neurology and Neurosurgery Headlines)
Source: Medscape Neurology and Neurosurgery Headlines - August 2, 2019 Category: Neurology Tags: Neurology & Neurosurgery News Source Type: news

Blast-mediated traumatic brain injury exacerbates retinal damage and amyloidosis in the APPswePSENd19e mouse model of Alzheimer's disease - Harper MM, Hedberg-Buenz A, Herlein J, Abrahamson EE, Anderson MG, Kuehn MH, Kardon RH, Poolman P, Ikonomovic MD.
PURPOSE: Traumatic brain injury (TBI) is a risk factor for developing chronic neurodegenerative conditions including Alzheimer's disease (AD). The purpose of this study was to examine chronic effects of blast TBI on retinal ganglion cells (RGC), optic nerv... (Source: SafetyLit)
Source: SafetyLit - June 29, 2019 Category: International Medicine & Public Health Tags: Burns, Electricity, Explosions, Fire, Scalds Source Type: news

Takeda scraps late-stage amyloidosis study
Takeda Pharmaceutical Co Ltd said on Wednesday it would discontinue a late-stage study testing its experimental treatment for amyloidosis, as it did not meet the first of two main goals. (Source: Reuters: Health)
Source: Reuters: Health - June 5, 2019 Category: Consumer Health News Tags: healthNews Source Type: news

TTR vs AL Cardiac Amyloidosis: Don't Rely on Imaging Alone TTR vs AL Cardiac Amyloidosis: Don't Rely on Imaging Alone
Drs Acharya, Grogan and Abou-Ezzeddine from Mayo discuss amyloid and TTR cardiac amyloidosis, with particular focus on diagnostic challenges and use of the pyrophosphate (PYP) scan.Mayo Clinic (Source: Medscape Cardiology Headlines)
Source: Medscape Cardiology Headlines - June 5, 2019 Category: Cardiology Tags: Cardiology Roundtable Source Type: news

Is Renal Infarction Linked to Cardiac Amyloidosis? Is Renal Infarction Linked to Cardiac Amyloidosis?
First study of its kind finds renal infarction in cardiac amyloidosis may be more common than previously thought, especially in patients with acute kidney injury, but an expert questions the results.Medscape Medical News (Source: Medscape Transplantation Headlines)
Source: Medscape Transplantation Headlines - June 5, 2019 Category: Transplant Surgery Tags: Nephrology News Source Type: news

AI+AR Equals Altoida ’s Alzheimer’s Detection Solution
Altoida is combining artificial intelligence, machine learning, and augmented reality to help detect Alzheimer’s disease. The Houston, TX-based company has struck a chord with venture capitalists, raising $6.3 million in a series A round. The financing was led by M Ventures, the corporate venture capital arm of the science and technology company Merck KGaA, Darmstadt, Germany, with participation from Grey Sky Venture Partners, VI Partners AG, Alpana Ventures, and FYRFLY Venture Partners. The new capital will be used to further expand Altoida's presence worldwide with an immediate focus on commercializati...
Source: MDDI - May 31, 2019 Category: Medical Devices Authors: Omar Ford Tags: Assembly and Automation Source Type: news

Renal infarction is associated with acute kidney injury in patients with cardiac amyloidosis
(Elsevier) Systemic amyloidosis is a major cause of renal injury, mostly due to direct kidney damage caused by deposits of abnormal protein called amyloid, in the kidney parenchyma. In patients with cardiac amyloidosis, renal infarction is associated with acute kidney injury according to a new study in Mayo Clinic Proceedings, published by Elsevier. Investigators recommend that a diagnosis of renal infarction should be systematically considered in patients with unexplained acute kidney injury in the context of cardiac amyloidosis. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - May 15, 2019 Category: International Medicine & Public Health Source Type: news

FDA Approves New Treatments Vyndaqel (tafamidis meglumine) and Vyndamax (tafamidis) for Heart Disease Caused by Transthyretin Mediated Amyloidosis
May 06, 2019 -- 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)... (Source: Drugs.com - New Drug Approvals)
Source: Drugs.com - New Drug Approvals - May 6, 2019 Category: Drugs & Pharmacology Source Type: news

FDA Approves Treatments for Heart Failure Caused by Rare Disease
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)
Source: Drugs.com - Pharma News - May 6, 2019 Category: Pharmaceuticals Source Type: news

Two Drugs Approved for Cardiomyopathy Caused by Amyloidosis
Two different versions of tafamadis get the FDA ’s nod to treat a rare heart disease.  (Source: ConsultantLive)
Source: ConsultantLive - May 6, 2019 Category: Internal Medicine Authors: Valerie DeBenedette Tags: Cardiology Cardiovascular Diseases Heart Health Latest e-News New Products The Latest Source Type: news

FDA OKs Two Tafamidis Formulations for Rare Cardiomyopathy FDA OKs Two Tafamidis Formulations for Rare Cardiomyopathy
These are the first FDA-approved treatments for transthyretin-mediated amyloidosis cardiomyopathy, a rare and often fatal disease caused by deposition of amyloid fibrils in the myocardium.FDA Approvals (Source: Medscape Cardiology Headlines)
Source: Medscape Cardiology Headlines - May 6, 2019 Category: Cardiology Tags: Cardiology News Alert Source Type: news

FDA approves new treatments for heart disease caused by a serious rare disease, transthyretin mediated amyloidosis
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)
Source: World Pharma News - May 6, 2019 Category: Pharmaceuticals Tags: Featured FDA Regulatory Affairs Source Type: news

Molecules that curb errant proteins of AL amyloidosis point to new type of therapy
(Scripps Research Institute) Scientists at Scripps Research have identified a group of small molecules that prevent structural changes to proteins that are at the root of AL amyloidosis, a progressive and often fatal disease. The drug mechanism that the team identified is analogous to a different drug that also originated at Scripps Research: Tafamidis, which stabilizes the protein transthyretin to treat the most common systemic amyloid disease that affects the heart or other organs. (Source: EurekAlert! - Cancer)
Source: EurekAlert! - Cancer - April 11, 2019 Category: Cancer & Oncology Source Type: news

Molecules that prevent protein misfolding point to new type of therapy for AL amyloidosis
(Scripps Research Institute) Scientists at Scripps Research have identified a group of small molecules that prevent structural changes to proteins that are at the root of AL amyloidosis, a progressive and often fatal disease. The drug mechanism that the team identified is analogous to a different drug that also originated at Scripps Research: Tafamidis, which stabilizes the protein transthyretin to treat the most common systemic amyloid disease that affects the heart or other organs. (Source: EurekAlert! - Cancer)
Source: EurekAlert! - Cancer - April 11, 2019 Category: Cancer & Oncology Source Type: news

On Rare Disease Day, Alnylam launches patient advocacy grant program
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)
Source: bizjournals.com Health Care:Pharmaceuticals headlines - February 28, 2019 Category: Pharmaceuticals Authors: Allison DeAngelis Source Type: news

C2N Launches Trial for Alzheimer ’s Detection Test
C2N Diagnostics is experiencing a couple of breakthroughs with its Alzheimer’s disease detection test - one courtesy of FDA. The agency, which is slowly rising back to full strength after the temporary government shutdown, granted the St. Louis-based company breakthrough device designation for a brain amyloidosis blood test. In addition, C2N said it is launching its Plasma Test for Amyloid Risk Screening (PARIS) Study that is evaluating and validating the clinical diagnostic performance of the C2N brain amyloidosis blood test. The company said the C2N test is a blood-based in vitro diagnostic being develo...
Source: MDDI - January 29, 2019 Category: Medical Devices Authors: Omar Ford Tags: IVD Source Type: news

Kidney Transplant May Be Appropriate for Some With AL Amyloidosis Kidney Transplant May Be Appropriate for Some With AL Amyloidosis
In selected patients with light chain (AL) amyloidosis who are reaching end-stage renal disease (ESRD), kidney transplantation is a worthwhile approach, according to Boston-based researchers.Reuters Health Information (Source: Medscape General Surgery Headlines)
Source: Medscape General Surgery Headlines - January 12, 2019 Category: Surgery Tags: Nephrology News Source Type: news

Quiz: Multiple Myeloma Patients With AL Amyloidosis
Challenge yourself with our latest quiz covering management of AL amyloidosis in the treatment of patients with multiple myeloma. (Source: CancerNetwork)
Source: CancerNetwork - December 10, 2018 Category: Cancer & Oncology Source Type: news

Localized Ureteral Amyloidosis Mimicking Urothelial Carcinoma Localized Ureteral Amyloidosis Mimicking Urothelial Carcinoma
How was the unusual origin of this patient's left-sided flank pain determined?Applied Radiology (Source: Medscape Today Headlines)
Source: Medscape Today Headlines - November 12, 2018 Category: Consumer Health News Tags: Radiology Journal Article Source Type: news

Alnylam shares take a hit amid sluggish sales of first drug
Shares of Alnylam Pharmaceuticals fell Wednesday after the Cambridge biotech reported a slow uptake of its first FDA-approved drug, an expensive, first-of-its-kind treatment for a rare disease that is difficult to diagnose. In August, Alnylam (Nasdaq: ALNY) became the first company to receive FDA approval for a drug using a new gene-silencing technology called RNA interference, or RNAi. The drug, Onpattro, treats a rare disease called hereditary ATTR amyloidosis that is hard to diagnose, which poses… (Source: bizjournals.com Health Care:Physician Practices headlines)
Source: bizjournals.com Health Care:Physician Practices headlines - November 7, 2018 Category: American Health Authors: Allison DeAngelis Source Type: news

New, more accessible staging system developed to predict survival for patients with AL Amyloidosis
(Boston Medical Center) A new staging system developed with a more accessible test to predict the chance of survival in patients living with light chain (AL) amyloidosis. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - November 7, 2018 Category: International Medicine & Public Health Source Type: news

FDA OKs Inotersen for Hereditary ATTR With Polyneuropathy FDA OKs Inotersen for Hereditary ATTR With Polyneuropathy
Inotersen treatment led to improvements in neurologic and quality-of-life endpoints in patients with hereditary transthyretin amyloidosis with polyneuropathy in a pivotal phase 3 trial.FDA Approvals (Source: Medscape Medical News Headlines)
Source: Medscape Medical News Headlines - October 8, 2018 Category: Consumer Health News Tags: Rheumatology News Alert Source Type: news

Akcea rebounds with FDA approval of first drug
After having one of its drugs rejected by the FDA in August, Cambridge biotech Akcea Therapeutics Inc. has turned the tide, receiving approval for another treatment for a rare genetic disease. The FDA approved Tegsedi, known generically as inotersen, an RNA-based drug that is designed to treat a rare and often fatal genetic disease called hereditary ATTR amyloidosis, or hATTR amyloidosis. According to Akcea (Nasdaq: AKCA), the drug works by slowing down production of a protein that can build up… (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - October 8, 2018 Category: Biotechnology Authors: Allison DeAngelis Source Type: news

Cambridge's Akcea rebounds with FDA approval of first drug
After having one of its drugs rejected by the FDA in August, Cambridge biotech Akcea Therapeutics Inc. turned the tide on Friday, receiving approval for another treatment for a rare genetic disease. The FDA approved Tegsedi, known generically as inotersen, an RNA-based drug that is designed to treat a rare and often fatal genetic disease called hereditary ATTR amyloidosis, or hATTR amyloidosis. According to Akcea (Nasdaq: AKCA), the drug works by slowing down production of a protein that can build… (Source: bizjournals.com Health Care:Physician Practices headlines)
Source: bizjournals.com Health Care:Physician Practices headlines - October 6, 2018 Category: American Health Authors: Allison DeAngelis Source Type: news

Cambridge's Akcea rebounds with FDA approval of first drug
After having one of its drugs rejected by the FDA in August, Cambridge biotech Akcea Therapeutics Inc. turned the tide on Friday, receiving approval for another treatment for a rare genetic disease. The FDA approved Tegsedi, known generically as inotersen, an RNA-based drug that is designed to treat a rare and often fatal genetic disease called hereditary ATTR amyloidosis, or hATTR amyloidosis. According to Akcea (Nasdaq: AKCA), the drug works by slowing down production of a protein that can build… (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - October 6, 2018 Category: Biotechnology Authors: Allison DeAngelis Source Type: news

FDA Approves Tegsedi (inotersen) for the Treatment of the Polyneuropathy of Hereditary Transthyretin-Mediated Amyloidosis
BOSTON and CARLSBAD, Calif., Oct. 05, 2018 (GLOBE NEWSWIRE) -- Akcea Therapeutics, Inc. (NASDAQ: AKCA), an affiliate of Ionis Pharmaceuticals, Inc., and Ionis Pharmaceuticals, Inc. (NASDAQ: IONS), announced today that the U.S. Food and Drug... (Source: Drugs.com - New Drug Approvals)
Source: Drugs.com - New Drug Approvals - October 5, 2018 Category: Drugs & Pharmacology Source Type: news

What Is to Blame for Cognitive Decline in Ageing? What Is to Blame for Cognitive Decline in Ageing?
What are the key drivers of cognitive decline in the elderly? A new study suggests that amyloidosis and neurodegeneration have both independent and synergistic effects.Brain (Source: Medscape Today Headlines)
Source: Medscape Today Headlines - September 17, 2018 Category: Consumer Health News Tags: Neurology & Neurosurgery Journal Article Source Type: news

What you missed on your summer vacation in Mass. biotech news
Here's a look at the Boston biotech news you missed during your summer vacation: Alnylam gets historic FDA approval for first drug Cambridge's Alnylam Pharmaceuticals (Nasdaq: ALNY) received FDA approval for its first drug on Aug. 10, a first-ever treatment for a rare genetic disease that uses RNA interference to silence genes. The drug treats hereditary ATTR amyloidosis, w hich causes the liver to protein abnormal proteins that impede nervous system and organ functions. Marketed under the name… (Source: bizjournals.com Health Care:Pharmaceuticals headlines)
Source: bizjournals.com Health Care:Pharmaceuticals headlines - September 4, 2018 Category: Pharmaceuticals Authors: Allison DeAngelis Source Type: news

Periodic Fevers: What to Watch For
(MedPage Today) -- Complications include amyloidosis and end-stage renal disease (Source: MedPage Today Pediatrics)
Source: MedPage Today Pediatrics - August 29, 2018 Category: Pediatrics Source Type: news

ESC: Tafamidis Cuts Deaths, Admissions in TTR Cardiac Amyloidosis
(MedPage Today) -- Plus clear advantage seen in 6-minute walk test (Source: MedPage Today Nephrology)
Source: MedPage Today Nephrology - August 27, 2018 Category: Urology & Nephrology Source Type: news

Alnylam gets FDA nod for first-ever RNAi drug
The U.S. FDA has approved Alnylam Pharmaceuticals ’ Onpattro, making it the first medicine specifically cleared to treat a rare and deadly disease called hereditary transthyretin amyloidosis. (Source: PharmaManufacturing.com)
Source: PharmaManufacturing.com - August 13, 2018 Category: Pharmaceuticals Source Type: news

FDA Approves Onpattro (patisiran) Targeted RNA-based Therapy for Polyneuropathy Caused by hATTR
August 10, 2018 -- The U.S. Food and Drug Administration today approved Onpattro (patisiran) infusion for the treatment of peripheral nerve disease (polyneuropathy) caused by hereditary transthyretin-mediated amyloidosis (hATTR) in adult patients.... (Source: Drugs.com - New Drug Approvals)
Source: Drugs.com - New Drug Approvals - August 10, 2018 Category: Drugs & Pharmacology Source Type: news

Patisiran Wins Two Firsts With FDA Approval
(MedPage Today) -- RNAi drug OK'd to treat polyneuropathy from hereditary transthyretin amyloidosis (Source: MedPage Today Primary Care)
Source: MedPage Today Primary Care - August 10, 2018 Category: Primary Care Source Type: news

Cambridge biotech Alnylam wins FDA approval for first drug
The FDA has approved Alnylam Pharmaceuticals Inc.'s first drug, a novel rare disease treatment that analysts believe could eventually top $1 billion in annual sales, marking a major milestone for one of the state's fastest-growing biotechs. The FDA said Friday it had approved Cambridge-based Alnylam's (Nasdaq: ALNY) drug patisiran, which targets a rare and often fatal genetic disease called hereditary ATTR amyloidosis, or hATTR amyloidosis. The disorder, which affects around 50,000 people worldwide,… (Source: bizjournals.com Health Care:Pharmaceuticals headlines)
Source: bizjournals.com Health Care:Pharmaceuticals headlines - August 10, 2018 Category: Pharmaceuticals Authors: Allison DeAngelis Source Type: news

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)
Source: Food and Drug Administration - August 10, 2018 Category: American Health Source Type: news

First RNAi Therapy Approved by FDA
Alnylam's patisiran interferes with the production of a mutated protein present in people with hereditary transthyretin amyloidosis. (Source: The Scientist)
Source: The Scientist - August 10, 2018 Category: Science Tags: News & Opinion Source Type: news