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 8, 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
