Rigshospitalet

Protara Therapeutics Announces Alignment with FDA on Registrational Path Forward for IV Choline Chloride in Patients Dependent on Parenteral Nutrition

Retrieved on: 
Venerdì, Aprile 5, 2024
Choline, Plasma, Liver, Nutritional science, Route of administration, Liver disease, Steatosis, Short bowel syndrome, Metabolism, Patient, Choline chloride, Cancer, ESPEN, Parenteral nutrition, Adolescence, Liver injury, Hepatology, Tubing (recreation), Fibrosis, Guideline, Society, Therapy, Copenhagen University Hospital, Safety, Pharmacokinetics, PN, FDA, ASPEN, Rigshospitalet, Food, Feeding tube, Extractive metallurgy

IV Choline Chloride has the potential to become the first FDA-approved IV formulation of choline for the 40,000 PN patients in the U.S.

Key Points: 
  • IV Choline Chloride has the potential to become the first FDA-approved IV formulation of choline for the 40,000 PN patients in the U.S.
  • The Company plans to advance the development of IV Choline Chloride as a source of choline for adult and adolescent patients on long-term PN.
  • The FDA has granted IV Choline Chloride Orphan Drug Designation for the prevention of choline deficiency in PN patients.
  • “We look forward to advancing the clinical development of IV Choline Chloride, which we believe has the potential to become the first FDA approved IV choline therapy for patients dependent on PN.

NorthSea Therapeutics Initiates Phase 2A Trial of Orziloben (NST-6179) in Intestinal Failure-Associated Liver Disease (IFALD)

Retrieved on: 
Mercoledì, Febbraio 21, 2024
Biotechnology, Pharmaceutical, Health, Clinical Trials, Liver, Collagen, Myofibroblast, PD, Liver disease, Cirrhosis, Learning, Short bowel syndrome, Conference, Hepatology, Liver failure, Safety, Steatosis, Rigshospitalet, PN, Therapy, Pharmacokinetics, Cholestasis, Fatty liver disease, Patient, ASPEN, Fibrosis, Disease, NASH, Pharmaceutical industry

The trial is a randomized, double-blind, Phase 2a, placebo-controlled study, which will be conducted at multiple sites across North America.

Key Points: 
  • The trial is a randomized, double-blind, Phase 2a, placebo-controlled study, which will be conducted at multiple sites across North America.
  • It is designed to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of Orziloben in adult subjects with IFALD.
  • Commenting on the milestone, Rob de Ree, NST’s CEO, said: "Dosing the first patient in our Phase 2a trial for Orziloben in IFALD is a significant achievement for NorthSea Therapeutics, and is a testament to our commitment to advance innovative treatments for liver diseases.
  • In one pre-clinical model of PN-induced liver injury, Orziloben treatment completely prevented severe cholestasis and the development of fibrosis.

Cybin to Host CYB003 Topline Depression Study Review and R&D Briefing on November 30, 2023, in New York City

Retrieved on: 
Lunedì, Novembre 20, 2023
Biotechnology, Mental Health, Health, Pharmaceutical, Clinical Trials, Psychopharmacology, Mental, Research, NYSE, MGH, Carlsberg Foundation, DSM-IV codes, Psychiatry, National academy, Dean (education), Endocrinology, Literature, European College of Neuropsychopharmacology, Book, Functional neuroimaging, Rigshospitalet, NEO, National Academy of Medicine, Medicine, University of Copenhagen, Neurology, Neuropharmacology, University, Epilepsy, Harvard Medical School, Human Brain Project, Letter, Society, Inc., Science, Hospital, Royal Danish Academy, Pharmaceutical industry, Depression

Dr. Fava is a world leader in the field of depression and clinical psychopharmacology.

Key Points: 
  • Dr. Fava is a world leader in the field of depression and clinical psychopharmacology.
  • Dr. Fava obtained his medical degree from the University of Padova School of Medicine and completed residency training in endocrinology at the same university.
  • He founded and was director of the hospital’s Depression Clinical and Research Program from 1990 until 2014.
  • Professor Knudsen received her medical and doctoral degrees from the University of Copenhagen, Denmark and completed her training in neurology.

First Clinical Results of ATA-100, a Gene Therapy for the Treatment of Limb-Girdle Muscular Dystrophy Type 2I/R9 (LGMD2I/R9), Presented at ESGCT

Retrieved on: 
Venerdì, Ottobre 27, 2023
Health, Genetics, Clinical Trials, Research, Science, Pharmaceutical, Biotechnology, Patient, FDA, United Kingdom, DSM-IV codes, IND, FKRP, International, Facial muscles, Conference, Cardiomyopathy, Weakness, Circulatory system, Data monitoring committee, Cell, Clinical trial, CNRS, Gene, ESGCT, Part, Rigshospitalet, Safety, European Society of Gene and Cell Therapy, Therapy, Muscular dystrophy, DSMB, Pharmaceutical industry, Vaccine, MD

ATA-100 is a one-time gene therapy for the treatment of fukutin-related protein (FKRP) limb-girdle muscular dystrophy Type 2I/R9 (LGMD2I/R9).

Key Points: 
  • ATA-100 is a one-time gene therapy for the treatment of fukutin-related protein (FKRP) limb-girdle muscular dystrophy Type 2I/R9 (LGMD2I/R9).
  • Atamyo also announced today that Data Safety Monitoring Board (DSMB) authorized the enrollment of the second dose cohort of the ATA-001 clinical trial.
  • ATA-100 is being evaluated in a multi-center phase 1b/2b in Denmark, France, and the United Kingdom, and has received IND clearance by FDA.
  • ATA-100, a single-administration gene therapy candidate for LGMD2I/R9, delivers a normal copy of the gene for production of FKRP proteins.

Atamyo Therapeutics Announces participation in Conferences and Key Scientific Communications on its LGMD programs

Retrieved on: 
Venerdì, Settembre 29, 2023
Science, Biotechnology, Research, Pharmaceutical, General Health, Health, Genetics, Clinical Trials, Muscular dystrophy, Natural history, International, Gene, C2, Rentschler, Part, ESGCT, Facial muscles, Neuromuscular disease, Diplomatic mission, Circulatory system, Rigshospitalet, Cell, Public, Oral, European Society of Clinical Microbiology and Infectious Diseases, Therapy, Conference, Pharmaceutical industry, Oyster farming, Nightclub, Rare-earth element, MD

Atamyo Therapeutics , a biotechnology company focused on the development of new-generation gene therapies targeting neuromuscular disease, today announced its participation to forthcoming conferences and scientific communications on its programs targeting limb-girdle muscular dystrophies (LGMDs).

Key Points: 
  • Atamyo Therapeutics , a biotechnology company focused on the development of new-generation gene therapies targeting neuromuscular disease, today announced its participation to forthcoming conferences and scientific communications on its programs targeting limb-girdle muscular dystrophies (LGMDs).
  • Stephane Degove, Atamyo CEO, will provide a corporate overview at two forthcoming conferences in October:
    Chardan's 7th Annual Genetic Medicines Conference, held in New York City:
    - Presentation on October 2, at 10:30 AM, in the Embassy room
    Cell & Gene Meeting on the Mesa, held in Carlsbad, CA, on October 10-12, 2023:
    - Presentation on October 10, at 5:15pm, at the Rentschler ATMP Ballroom
    Two upcoming communications at the 28th Annual Congress of the World Muscle Society (3-7 October 2023 in Charleston, SC)
    • Oral Presentation by A. Brureau (Genethon), at Ballroom C2 on October 4, 2023 from 18h15 to 18h45
    • Poster Board Number 282 at Ballroom C, on October 4, 2023, 17h15-18h15
    Natural history of limb girdle muscular dystrophy R9: one-year follow-up of a European cohort
    • Oral Presentation by J. Vissing (Copenhagen Neuromuscular Center, Rigshospitale) at Ballroom C2 on October 4, 2023 from 18h15 to 18h45
    • Poster Board Number 283 at Ballroom C, on October 4, 2023, 17h15-18h15
    Two communications describing Atamyo’s LGMD programs will be presented at the 30th Annual Congress of the European Society of Gene & Cell Therapy (ESGCT) that will take place on October 24-27, 2023 in Brussel (Belgium):
    • Oral presentation by Nicolai Preisler, MD, Rigshospitalet, Copenhagen, during the Cardiovascular and muscular diseases session, on Friday October 27, 11h30-13h00 – Le BEL, Tour & Taxis
    Oral presentation on Atamyo’s LGMD-R9 program at the 2023 International Limb-Girdle Muscular Dystrophy Conference (28-29 October 2023, Washington DC)
    • Oral presentation by John Vissing, MD, Rigshospitalet, Copenhagen, during the Clinical Trials Updates (Part 1) Session, on October 29, 11h00am-12h10pm – Constitution Ballroom, Grand Hyatt

Heart Centre Rigshospitalet Enhances Leadership in Patient Care with Adoption of Latest Robotic Technology

Retrieved on: 
Mercoledì, Settembre 6, 2023
Partnership, Copenhagen University Hospital, Heart failure, Arrest, Stroke, Genesis, Arrhythmia, Robotic magnetic navigation, Hospital, Patient, Physician, Risk, Rigshospitalet, Health, NYSE, Shahid Gangalal National Heart Centre, Safety, Electrophysiology, Medical device, Nursing, Stereotaxis

The Genesis system is the latest and most advanced robotic technology available for the treatment of heart rhythm disorders.

Key Points: 
  • The Genesis system is the latest and most advanced robotic technology available for the treatment of heart rhythm disorders.
  • Electrophysiologists at The Heart Centre Rigshospitalet were early pioneers, and are global leaders, in the use of advanced robot technology for the treatment of cardiac arrhythmias.
  • Since installation of the first robotic system in 2006, Rigshospitalet physicians have successfully treated over 5,000 patients using Stereotaxis’ robotic technology.
  • “We look forward to continuing a strong collaboration that advances patient care, clinical science, and technology development in electrophysiology.”

Heart Failure start-up, Acorai, embarks on the next vital stage towards regulatory approval

Retrieved on: 
Mercoledì, Agosto 16, 2023
Well, Duke University, Royal Brompton and Harefield NHS Foundation Trust, Partnership, De novo, Heart failure, Therapy, Sahlgrenska University Hospital, Prevalence, Health care, MLG, Harefield Hospital, Quality of life, Intracardiac injection, Oct, History, Teaching hospital, TCT, Duke University School of Medicine, Royal Brompton Hospital, Vital signs, Patient, Machine learning, Rigshospitalet, Algorithm, Pedigree, Mid America Heart Institute, Entrepreneurship, Coronary artery disease, FDA, City centre, Health, ICPM, Luke the Evangelist, St Luke's, Software, Medical device, Pharmaceutical industry, Kansas City metropolitan area

Acorai was designated as a breakthrough device by the US FDA in August 2023 and they aim to submit their heart monitor for regulatory approval to the FDA and CE-marking in 2024.

Key Points: 
  • Acorai was designated as a breakthrough device by the US FDA in August 2023 and they aim to submit their heart monitor for regulatory approval to the FDA and CE-marking in 2024.
  • Acorai is developing a first-of-its-kind, scalable medical device for non-invasive intracardiac pressure monitoring to improve heart failure management for more than 64 million patients worldwide.
  • Dr. Dar is a cardiologist specializing in heart failure and leads the advanced heart failure, heart transplant, and mechanical circulatory support research programme at Harefield Hospital.
  • We look forward to collaborating with Acorai, whose heart monitor may have the potential to 'change the game' in diagnostics, monitoring, and treatment of patients with heart failure."

Heart Failure start-up, Acorai, embarks on the next vital stage towards regulatory approval

Retrieved on: 
Mercoledì, Agosto 16, 2023
Well, Duke University, Royal Brompton and Harefield NHS Foundation Trust, Partnership, De novo, Heart failure, Therapy, Sahlgrenska University Hospital, Prevalence, Health care, MLG, Harefield Hospital, Quality of life, Intracardiac injection, Oct, History, Teaching hospital, TCT, Duke University School of Medicine, Royal Brompton Hospital, Vital signs, Patient, Machine learning, Rigshospitalet, Algorithm, Pedigree, Mid America Heart Institute, Entrepreneurship, Coronary artery disease, FDA, City centre, Health, ICPM, Luke the Evangelist, St Luke's, Software, Medical device, Pharmaceutical industry, Kansas City metropolitan area

Acorai was designated as a breakthrough device by the US FDA in August 2023 and they aim to submit their heart monitor for regulatory approval to the FDA and CE-marking in 2024.

Key Points: 
  • Acorai was designated as a breakthrough device by the US FDA in August 2023 and they aim to submit their heart monitor for regulatory approval to the FDA and CE-marking in 2024.
  • Acorai is developing a first-of-its-kind, scalable medical device for non-invasive intracardiac pressure monitoring to improve heart failure management for more than 64 million patients worldwide.
  • Dr. Dar is a cardiologist specializing in heart failure and leads the advanced heart failure, heart transplant, and mechanical circulatory support research programme at Harefield Hospital.
  • We look forward to collaborating with Acorai, whose heart monitor may have the potential to 'change the game' in diagnostics, monitoring, and treatment of patients with heart failure."

Octapharma Puts a Spotlight on the Infection Burden in Patients With Chronic Lymphocytic Leukaemia (CLL) at the European Hematology Association (EHA) Hybrid Congress 2023

Retrieved on: 
Mercoledì, Giugno 7, 2023
Oncology, Health, Other Science, Research, Pharmaceutical, Science, CLL, Infection, Immunodeficiency, University, Victorian Comprehensive Cancer Centre, University of Padua, COVID-19, Clinical trial, Risk, SID, Congress, Death, Medical director, Patient, Guideline, Rigshospitalet, Immunoglobulin G, Glossary of education terms (G–L), Disease, Hypogammaglobulinemia, Safety, Pharmaceutical industry, Medicine, Octapharma

Secondary immunodeficiency (SID) is a common complication in patients with haematological malignancies such as CLL.

Key Points: 
  • Secondary immunodeficiency (SID) is a common complication in patients with haematological malignancies such as CLL.
  • Finally, Professor Livio Trentin, University of Padua, Italy, will speak about his experience using subcutaneous immunoglobulin therapy for infection prophylaxis in patients with CLL.
  • Stephan Stilgenbauer, Professor of Medicine and Medical Director of the Comprehensive Cancer Centre in Ulm, Germany, will chair the session.
  • The results of this analysis on 3,846 SID patients will be displayed and commented during the poster session of the Congress on June 9, 2023:

AMGEN PRESENTS NEW RESEARCH ON OTEZLA® (APREMILAST) IN PSORIATIC ARTHRITIS AT EULAR 2023

Retrieved on: 
Martedì, Maggio 30, 2023
Apremilast, Inflammation, AMGN, Amgen, Psoriatic arthritis, University, Arthritis, University of Copenhagen, Ligament, Synovitis, Clinical trial, Finger, HDL, Research, Congress, MRI, BME, Magnetic resonance imaging, HIV disease progression rates, Poster, European, Rigshospitalet, LDL, Tenosynovitis, Joint, Global Medical Device Nomenclature, Body mass index, EULAR, All India Mahila Congress, Tendon, Glycated hemoglobin, Bone erosion, Pharmaceutical industry, Medical imaging, Silviculture, Hand, Rheumatology, Patient, MOSAIC

THOUSAND OAKS, Calif., May 30, 2023 /PRNewswire/ -- Amgen (NASDAQ:AMGN) today announced new research examining the use of Otezla® (apremilast) in psoriatic arthritis, including the Phase 4 MOSAIC study and an exploratory analysis of cardiometabolic risk factors, which are commonly elevated in patients with psoriatic disease. The findings will be presented at the 2023 European Congress of Rheumatology (EULAR), taking place May 31-June 3 in Milan, Italy.

Key Points: 
  • The findings will be presented at the 2023 European Congress of Rheumatology (EULAR), taking place May 31-June 3 in Milan, Italy.
  • "Research presented at EULAR sheds new light on psoriatic arthritis and the role of our oral medication Otezla," said Ponda Motsepe-Ditshego, vice president, Global Medical at Amgen.
  • MOSAIC evaluated Otezla's effect on joint inflammation and structural progression of psoriatic arthritis measured by MRI.
  • "The results of this study are encouraging, as they provide important insights about Otezla treatment and its efficacy on both clinical and inflammatory manifestations of psoriatic arthritis."