Polyneuropathy

NeuroMetrix Announces Publication of Study Demonstrating that DPNCheck Accurately Diagnoses Diabetic Peripheral Neuropathy

Retrieved on: 
Thursday, March 14, 2024
Diagnosis, Hayashi, DPN, Diabetes, Patient, Dunker, Polyneuropathy, EKG

WOBURN, Mass., March 14, 2024 (GLOBE NEWSWIRE) -- NeuroMetrix, Inc. (Nasdaq: NURO) noted the recent publication of a large study demonstrating that DPNCheck® combined with standard EKG or DPNCheck alone accurately detects diabetic peripheral neuropathy (DPN).

Key Points: 
  • WOBURN, Mass., March 14, 2024 (GLOBE NEWSWIRE) -- NeuroMetrix, Inc. (Nasdaq: NURO) noted the recent publication of a large study demonstrating that DPNCheck® combined with standard EKG or DPNCheck alone accurately detects diabetic peripheral neuropathy (DPN).
  • The reference standard for DPN was a gold standard nerve conduction study.
  • DPNCheck combined with CVR-R had high diagnostic performance with an area under the receiver operating curve of 0.88.
  • The authors concluded "The use of DPNCheck and electrocardiogram would make the diagnosis of DPN simple, ubiquitous, and with high reproducibility and reliability."

Eplontersen granted U.S. FDA Fast Track designation for patients with transthyretin-mediated amyloid cardiomyopathy

Retrieved on: 
Thursday, February 8, 2024
Ionis Pharmaceuticals, RNA, Cardiovascular disease, Therapy, Fast Track, Patient, FDA, Orphan, Amyloidosis, IONS, AstraZeneca, Food, Ionis, ATTR, Polyneuropathy, DSM-IV codes, Pharmaceutical industry

CARLSBAD, Calif., Feb. 8, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) announced today that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to Ionis and AstraZeneca's eplontersen, an investigational therapy for the treatment of transthyretin-mediated amyloid cardiomyopathy (ATTR-CM) in adults.

Key Points: 
  • CARLSBAD, Calif., Feb. 8, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) announced today that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to Ionis and AstraZeneca's eplontersen, an investigational therapy for the treatment of transthyretin-mediated amyloid cardiomyopathy (ATTR-CM) in adults.
  • The FDA grants development programs Fast Track designation to facilitate the development and expedite the review of drugs that demonstrate the potential to treat serious conditions and fill an unmet medical need.
  • "CARDIO-TTRansform is the largest, most comprehensive study ever conducted in ATTR-CM patients, with results expected as early as next year."
  • WAINUA was granted Orphan Drug Designation in the U.S. and in the EU for the treatment of transthyretin-mediated amyloidosis (ATTR).

Intellia Therapeutics Highlights its Three-Year Strategic Priorities and Anticipated 2024 Key Milestones

Retrieved on: 
Thursday, January 4, 2024
Liver, Conference, Science, Gene editing, Biotechnology Innovation Organization, Amyloidosis, BIO, CRISPR, Phase 1, Tissue, DNA, Cardiomyopathy, Therapy, ATTR, Intellia Therapeutics, NTLA, Clinical trial, Polyneuropathy, NK, AATD, Lung, Hereditary angioedema, Webcast, Patient, Medicine, HAE, BLA, Pharmaceutical industry

Organizational Streamlining: Following an internal strategic review, Intellia will be streamlining company-wide operations to further focus resources on key strategic priorities and programs.

Key Points: 
  • Organizational Streamlining: Following an internal strategic review, Intellia will be streamlining company-wide operations to further focus resources on key strategic priorities and programs.
  • Cash Runway: Intellia ended the fourth quarter of 2023 with approximately $1.0 billion in cash, cash equivalents and marketable securities.
  • Prepare for the Phase 3 study of NTLA-2001 for the treatment of ATTR amyloidosis with polyneuropathy (ATTRv-PN) in 2024.
  • A live webcast will be available through the Events and Presentations page of the Investors & Media section on Intellia’s website, www.intelliatx.com .

Orsini selected as the exclusive specialty pharmacy for WAINUATM (eplontersen) for adults with hereditary transthyretin-mediated amyloid polyneuropathy

Retrieved on: 
Thursday, January 4, 2024
Vitamin A deficiency, ELK, AstraZeneca, IDN, FDA, Disease, Ionis, Orsini, Serum, Polyneuropathy, Internationalized domain name, Vitamin A, Pharmacy, Patient, Diagnosis, Disability studies, Amyloidosis

ELK GROVE VILLAGE, Ill., Jan. 4, 2024 /PRNewswire/ -- Orsini Specialty Pharmacy ("Orsini") has been selected by AstraZeneca and Ionis as the exclusive specialty pharmacy partner for WAINUA™ (eplontersen), an FDA-approved treatment for adults living with hereditary transthyretin-mediated amyloid polyneuropathy (commonly referred to as hATTR-PN or ATTRv-PN).

Key Points: 
  • ELK GROVE VILLAGE, Ill., Jan. 4, 2024 /PRNewswire/ -- Orsini Specialty Pharmacy ("Orsini") has been selected by AstraZeneca and Ionis as the exclusive specialty pharmacy partner for WAINUA™ (eplontersen), an FDA-approved treatment for adults living with hereditary transthyretin-mediated amyloid polyneuropathy (commonly referred to as hATTR-PN or ATTRv-PN).
  • "Orsini is proud to be the exclusive specialty pharmacy offering access to WAINUA as a new treatment option for patients living with ATTRv-PN," Orsini CEO Brandon Tom said.
  • Indication: WAINUA injection, for subcutaneous use, 45 mg is indicated for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults.
  • Note: While Orsini is the exclusive specialty pharmacy partner for WAINUA, it may also be available for use by integrated delivery networks (IDNs) within qualifying IDN-owned specialty pharmacies.

ORYZON Awarded with a Grant for ORY-4001 From the ALS Association in the U.S.

Retrieved on: 
Monday, December 4, 2023
ALS, Peripheral neuropathy, Toxicology, HDAC6, Research, Polyneuropathy, Disease, IPSC, CMT, ALS Association, Life expectancy, Respiratory failure, USD, Genomics, Death, Systemic disease, Proteostasis, Neurodegenerative disease, Axonal transport, Diagnosis, Safety, Pharmaceutical industry

The Association has awarded Oryzon a 498,690 USD grant through its Lawrence and Isabel Barnett Drug Development Program to support the regulatory preclinical development of ORY-4001, a highly selective HDAC6 inhibitor, for amyotrophic lateral sclerosis (ALS).

Key Points: 
  • The Association has awarded Oryzon a 498,690 USD grant through its Lawrence and Isabel Barnett Drug Development Program to support the regulatory preclinical development of ORY-4001, a highly selective HDAC6 inhibitor, for amyotrophic lateral sclerosis (ALS).
  • Dr. Jordi Xaus, Oryzon’s CSO, said: “It is an honor to have the support of the ALS Association.
  • Established in 1985, the ALS Association is the largest philanthropic funder of ALS research in the world.
  • The Association is working to make ALS a livable disease while urgently searching for new treatments and a cure.

WAINUA™ (eplontersen) Granted First-Ever Regulatory Approval in the US for the Treatment of Adults With Polyneuropathy of Hereditary Transthyretin-Mediated Amyloidosis

Retrieved on: 
Friday, December 22, 2023
Research, FDA, Neurology, Genetics, Clinical Trials, Biotechnology, Health, Pharmaceutical, Science, Quality of life, Tissue, Heart, Heredity, Serum, Polyneuropathy, Death, Inotersen, Ageing, TTR, JAMA, Vitamin A, Diagnosis, Metabolism, Amyloidosis, Pancreas, Disease, Safety, Journal, Heart failure, Disability studies, Sympathetic nervous system, Vitamin A deficiency, Food, Biopharmaceutical, AstraZeneca, Caregiver, ATTR, Patient, Medicine, LICA, Ionis, American Medical Association, Growth, Central nervous system, Pharmaceutical industry

It is also a pivotal moment for Ionis as WAINUA will be the first in a steady cadence of potential commercial launches for the company.

Key Points: 
  • It is also a pivotal moment for Ionis as WAINUA will be the first in a steady cadence of potential commercial launches for the company.
  • Reduced Serum Vitamin A Levels and Recommended Supplementation WAINUA leads to a decrease in serum vitamin A levels.
  • Refer patient to an ophthalmologist if ocular symptoms suggestive of vitamin A deficiency occur.
  • Please see link to US Full Prescribing Information for WAINUA.

WAINUA™ (eplontersen) granted regulatory approval in the U.S. for the treatment of adults with polyneuropathy of hereditary transthyretin-mediated amyloidosis

Retrieved on: 
Thursday, December 21, 2023
Quality of life, LICA, Food, AstraZeneca, Caregiver, Ionis Pharmaceuticals, FDA, Amyloidosis, Disease, ATTR, Serum, Polyneuropathy, Death, American Medical Association, Disability studies, TTR, JAMA, Patient, Diagnosis, IONS, Medicine, Journal, Heart failure, Neurology, Pharmaceutical industry

WAINUA is the only approved medicine for the treatment of ATTRv-PN that can be self-administered via an auto-injector.

Key Points: 
  • WAINUA is the only approved medicine for the treatment of ATTRv-PN that can be self-administered via an auto-injector.
  • "Approval of WAINUA represents a meaningful advancement in treatment, one that gives those who are living with transthyretin-mediated amyloid polyneuropathy help managing the disease."
  • WAINUA is a ligand-conjugated antisense oligonucleotide (LICA) medicine designed to reduce the production of TTR protein at its source.
  • "There is an urgent medical need for new therapies for people living with hereditary transthyretin-mediated amyloid polyneuropathy," said Ruud Dobber, executive vice president, BioPharmaceuticals Business Unit, AstraZeneca.

Alnylam Highlights Significant Progress with Platform Innovation and Clinical Pipeline at R&D Day

Retrieved on: 
Wednesday, December 13, 2023
Biotechnology, Health, Genetics, Pharmaceutical, Clinical Trials, CNS, Hypertension, Cerebral amyloid angiopathy, Hepatocellular carcinoma, Gene, ALN, Cardiomyopathy, RNA interference, IND, Tissue, Liver, Sanofi, Amyloidosis, Gene silencing, Overweight, KHK, Patient, FGF21, CAA, Part, Obesity, T2DM, Neoplasm, Investigational New Drug, Therapy, LNP, Development, Shanda, Growth, HCC, Central nervous system, Type, TTR, Immunotherapy, Polyneuropathy, Haemophilia, Medication, Biology, Coagulopathy, Traffic barrier, Adipose tissue, Vaccine, Medical device, Pharmaceutical industry, Video game, Alnylam Pharmaceuticals, Capella, ATTR

We expect our track record of strong pipeline and commercial execution to continue through the end of 2025 and beyond,” said Akshay Vaishnaw, M.D., Ph.D., Chief Innovation Officer of Alnylam.

Key Points: 
  • We expect our track record of strong pipeline and commercial execution to continue through the end of 2025 and beyond,” said Akshay Vaishnaw, M.D., Ph.D., Chief Innovation Officer of Alnylam.
  • In addition, Alnylam will share an update on delivery advances driving ALN-BCAT, which will soon begin clinical testing in patients with hepatocellular carcinoma (HCC).
  • A peak mean TTR reduction of 97% was achieved at Day 29 and a mean TTR reduction of 93% was sustained at Day 180.
  • The Company’s R&D Day event will be held today, Wednesday, Dec. 13, from 8:30 a.m. to 12:45 p.m.

Intellia Presents New Interim Data from the Ongoing Phase 1 Study of NTLA-2001 at the 4th International ATTR Amyloidosis Meeting

Retrieved on: 
Thursday, November 2, 2023
Amyloid, FDA, Standard of care, Headache, IND, International, ATTR, Cardiomyopathy, Diarrhea, CRISPR, Intellia Therapeutics, TTR, Disease, Polyneuropathy, Serum, Amyloidosis, Safety, Therapy, Patient, Back pain, NTLA, Toxic leukoencephalopathy, Pharmaceutical industry, Vaccine

Results were presented in an oral presentation at the 4th International ATTR Amyloidosis Meeting, held Nov. 2–3 in Madrid, Spain.

Key Points: 
  • Results were presented in an oral presentation at the 4th International ATTR Amyloidosis Meeting, held Nov. 2–3 in Madrid, Spain.
  • “With 65 patients reported from the Phase 1 study, this update represents the largest clinical dataset for an in vivo CRISPR-based investigational therapy.
  • These positive interim results add to the growing body of data that demonstrates deep and durable reductions of serum TTR after a single dose of NTLA-2001.
  • The Phase 1 trial is a two-part study evaluating NTLA-2001 in patients with either ATTR amyloidosis with cardiomyopathy (ATTR-CM) or hereditary ATTR amyloidosis with polyneuropathy (ATTRv-PN).

Alnylam Pharmaceuticals Reports Third Quarter 2023 Financial Results and Highlights Recent Period Activity

Retrieved on: 
Thursday, November 2, 2023
Biotechnology, Pharmaceutical, Health, Clinical Trials, FDA, ATTR, The New England Journal of Medicine, Science, Therapy, Bachelor of Medicine, Bachelor of Surgery, Cardiomyopathy, Medication, Use, Viral hepatitis, Symantec Endpoint Protection, Conference, American Heart Association, Quarter, Acquisition, Haemophilia, Clinical trial, TTR, Truly Madly Deeply – Ultra Rare Tracks, Growth, Polyneuropathy, Risk, Hypertension, RNA interference, ALN, Roche, Sanofi, Amyloidosis, Patient, APOLLO, ASCVD, Phase 1, Pharmaceutical industry, Video game, Medical device, Aluminium, Regeneron Pharmaceuticals, Alnylam Pharmaceuticals, Inclisiran

Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today reported its consolidated financial results for the third quarter ended September 30, 2023 and reviewed recent business highlights.

Key Points: 
  • Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today reported its consolidated financial results for the third quarter ended September 30, 2023 and reviewed recent business highlights.
  • Attained over 3,790 hATTR amyloidosis patients with polyneuropathy worldwide on commercial treatment with ONPATTRO or AMVUTTRA as of September 30, 2023.
  • Alnylam announces today that it will present a review of its RNAi platform and pipeline progress at a virtual R&D Day on December 13, 2023.
  • Financial Results for the Quarter Ended September 30, 2023