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

FDA approves first-of-its kind targeted RNA-based therapy to treat a rare disease
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. This is the first FDA-approved treatment for patients with polyneuropathy caused by hATTR, a rare, debilitating and often fatal genetic disease characterized by the buildup of abnormal amyloid protein in peripheral nerves, the heart and other organs. (Source: World Pharma News)
Source: World Pharma News - August 10, 2018 Category: Pharmaceuticals Tags: Featured FDA Regulatory Affairs Source Type: news

First Case of Pulmonary Amyloidosis in a Dental Technician First Case of Pulmonary Amyloidosis in a Dental Technician
A dental technician developed multiple granulomas with amyloid deposition in the lungs after occupational exposure to silica. What can we learn from this rare case?BMC Pulmonary Medicine (Source: Medscape Today Headlines)
Source: Medscape Today Headlines - August 2, 2018 Category: Consumer Health News Tags: Pulmonary Medicine Journal Article Source Type: news

New Agents Show Promise for Severe and Fatal Genetic Disease New Agents Show Promise for Severe and Fatal Genetic Disease
Hereditary transthyretin amyloidosis is caused by mutation in the TTR gene. Two promising new agents may slow the progression and improve the symptoms of this progressive and fatal disease.Medscape Medical News (Source: Medscape Medical News Headlines)
Source: Medscape Medical News Headlines - July 10, 2018 Category: Consumer Health News Tags: Neurology & Neurosurgery News Source Type: news

Patisiran, Inotersen Aid Hereditary Transthyretin Amyloidosis
THURSDAY, July 5, 2018 -- For patients with hereditary transthyretin amyloidosis with polyneuropathy, an investigational RNA interference therapeutic agent (patisiran) and a 2'-O-methoxyethyl-modified antisense oligonucleotide (inotersen), which... (Source: Drugs.com - Pharma News)
Source: Drugs.com - Pharma News - July 5, 2018 Category: Pharmaceuticals Source Type: news

Oligonucleotide Drugs Show Promise in Transthyretin Amyloidosis (CME/CE)
(MedPage Today) -- Patisiran, inotersen show peripheral neuropathy progression can be slowed (Source: MedPage Today Neurology)
Source: MedPage Today Neurology - July 5, 2018 Category: Neurology Source Type: news

New England Journal of Medicine Publishes Results from Pivotal Study of TEGSEDI(TM) (inotersen) for the Treatment of Hereditary ATTR Amyloidosis
TEGSEDI treatment demonstrated substantial improvement in measures of neuropathy progression and quality of life CARLSBAD, Calif., and CAMBRIDGE, Mass., July 5, 2018 -- (Healthcare Sales & Marketing Network) -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS... Biopharmaceuticals, Neurology Ionis Pharmaceuticals, Akcea Therapeutics, TEGSEDI, inotersen, hATTR (Source: HSMN NewsFeed)
Source: HSMN NewsFeed - July 5, 2018 Category: Pharmaceuticals Source Type: news

Alliance Medical Group acquires Piramal Imaging
The Alliance Medical Group has reached an agreement to acquire Piramal Imaging,...Read more on AuntMinnie.comRelated Reading: Are amyloid PET scans ready for the clinical setting? PET with novel tracers foretells early Alzheimer's risk Piramal partner gets Canadian approval for NeuraCeq JNM: Florbetaben-PET detects cardiac amyloidosis Piramal Imaging inks Taiwan deal (Source: AuntMinnie.com Headlines)
Source: AuntMinnie.com Headlines - July 2, 2018 Category: Radiology Source Type: news

Are amyloid PET scans ready for the clinical setting?
The use of PET scans to confirm the presence of dementia-inducing amyloid plaque...Read more on AuntMinnie.comRelated Reading: PET links amyloid, vascular factors to cognitive decline PET with novel tracers foretells early Alzheimer's risk PiB-PET study strengthens link between amyloid, dementia High amyloid levels on PET may indicate early Alzheimer's JNM: Florbetaben-PET detects cardiac amyloidosis (Source: AuntMinnie.com Headlines)
Source: AuntMinnie.com Headlines - June 15, 2018 Category: Radiology Source Type: news

CHMP Recommends Approval for Inotersen in hATTR CHMP Recommends Approval for Inotersen in hATTR
Inotersen has won recommendation of the EMA's Committee for Medicinal Products for Human Use for treatment of hereditary transthyretin amyloidosis.International Approvals (Source: Medscape Medical News Headlines)
Source: Medscape Medical News Headlines - June 7, 2018 Category: Consumer Health News Tags: Neurology & Neurosurgery News Alert Source Type: news

Lifetime Risks Temper Biomarker Tests for Alzheimer Dz Dementia
Those younger than 85 years with MCI, amyloidosis, neurodegeneration have risk>50 percent (Source: The Doctors Lounge - Psychiatry)
Source: The Doctors Lounge - Psychiatry - June 7, 2018 Category: Psychiatry Tags: Neurology, Pathology, Psychiatry, Journal, Source Type: news

AD Link Between APOE, Tau Stronger in Women
(MedPage Today) -- But no association seen with amyloidosis or tangles (Source: MedPage Today Neurology)
Source: MedPage Today Neurology - May 9, 2018 Category: Neurology Source Type: news

APOLLO: Patisiran Reduces Polyneuropathy in hATTR Amyloidosis APOLLO: Patisiran Reduces Polyneuropathy in hATTR Amyloidosis
Randomized data from the APOLLO phase 3 study show significant improvement in polyneuropathy with treatment,'the first time in the amyloid story,'researchers say.Medscape Medical News (Source: Medscape Medical News Headlines)
Source: Medscape Medical News Headlines - April 26, 2018 Category: Consumer Health News Tags: Neurology & Neurosurgery News Source Type: news

Woodford-backed biotech Prothena plunges as key drug fails
LONDON (Reuters) - Shares in Prothena, a biotech company backed by British investor Neil Woodford, plunged 70 percent on Monday after its main drug for treating a rare disease called AL amyloidosis failed in a crucial clinical trial. (Source: Reuters: Health)
Source: Reuters: Health - April 23, 2018 Category: Consumer Health News Tags: healthNews Source Type: news

Eidos Therapeutics completes $64M Series B financing
Funding supports Phase 2 clinical trials of AG10, a potential best-in-class therapy for transthyretin amyloidosis, and ongoing preparation for Phase 3 clinical trials SAN FRANCISCO, April 5, 2018 -- (Healthcare Sales & Marketing Network) -- Eidos Thera... Biopharmaceuticals, Venture Capital Eidos Therapeutics, transthyretin amyloidosis (Source: HSMN NewsFeed)
Source: HSMN NewsFeed - April 5, 2018 Category: Pharmaceuticals Source Type: news

Case: Breast Amyloidosis Detected With 3D Mammography Case: Breast Amyloidosis Detected With 3D Mammography
Review the imaging characteristics of breast amyloidosis in this unusual case. How should definitive diagnosis be made?Applied Radiology (Source: Medscape Today Headlines)
Source: Medscape Today Headlines - February 6, 2018 Category: Consumer Health News Tags: Radiology Journal Article Source Type: news

Alnylam Announces FDA Acceptance of New Drug Application (NDA) and Priority Review Status for Patisiran, an Investigational RNAi Therapeutic for the Treatment of Hereditary ATTR (hATTR) Amyloidosis
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Feb. 1, 2018-- Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today that the U.S. Food and Drug Administration (FDA) has accepted for filing its New Drug Application... (Source: Drugs.com - New Drug Applications)
Source: Drugs.com - New Drug Applications - February 1, 2018 Category: Drugs & Pharmacology Source Type: news

Alnylam Completes Submission of New Drug Application to U.S. Food and Drug Administration (FDA) for Patisiran for the Treatment of Hereditary ATTR (hATTR) Amyloidosis
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Dec. 12, 2017-- Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today the completion of the rolling submission of a New Drug Application (NDA) to the U.S. Food and... (Source: Drugs.com - New Drug Applications)
Source: Drugs.com - New Drug Applications - December 12, 2017 Category: Drugs & Pharmacology Source Type: news

U.S. Food and Drug Administration (FDA) Grants Alnylam Breakthrough Therapy Designation (BTD) for Patisiran for the Treatment of Hereditary ATTR (hATTR) Amyloidosis with Polyneuropathy
CAMBRIDGE, Mass.--(BUSINESS WIRE) November 20, 2017 -- Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation... (Source: Drugs.com - New Drug Applications)
Source: Drugs.com - New Drug Applications - November 20, 2017 Category: Drugs & Pharmacology Source Type: news

Alnylam Initiates Rolling Submission of New Drug Application (NDA) to U.S. Food and Drug Administration (FDA) for Patisiran for the Treatment of Hereditary ATTR (hATTR) Amyloidosis
CAMBRIDGE, Mass.--(BUSINESS WIRE) November 16, 2017 -- Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced the company has initiated submission of a rolling New Drug Application (NDA) to the U.S. Food... (Source: Drugs.com - New Drug Applications)
Source: Drugs.com - New Drug Applications - November 16, 2017 Category: Drugs & Pharmacology Source Type: news

Alnylam shares hit all-time high as rare disease drug continues to impress
Alnylam Pharmaceuticals added more than $1.5 billion to its market cap on Thursday after unveiling new data from a closely watched trial of its lead rare disease drug, which appeared to outperform a competitor ’s drug targeting the same disorder. Alnylam (Nasdaq: ALNY) reported new details from a Phase 3 trial of patisiran, a potential treatment for a disease called amyloidosis ­that affects around 10,000 people. The company previously said in September that the study had met all of its goals,… (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - November 2, 2017 Category: Biotechnology Authors: Max Stendahl Source Type: news