MCID

4DMT Presents Interim Data from 4D-310 INGLAXA Phase 1/2 Clinical Trials for Fabry Disease Cardiomyopathy at WORLDSymposium™ 2024

Retrieved on: 
Monday, February 12, 2024
C102, Atypical hemolytic uremic syndrome, Fabry disease, CPET, VO2, FDA, Quality of life, Cardiovascular disease, Data, GLS, Safety, MCID, Overalls, Death, Patient, Echocardiography, Chelsea Handler, Pharmaceutical industry

The data was presented in a late-breaking session at WORLDSymposium™ 2024 in San Diego, California on Friday, February 9, 2024.

Key Points: 
  • The data was presented in a late-breaking session at WORLDSymposium™ 2024 in San Diego, California on Friday, February 9, 2024.
  • “We are pleased to see 4D-310 continue to consistently demonstrate clinical activity across multiple important cardiac endpoints including cardiac function, exercise capacity and quality of life,” said Robert Kim, M.D., Chief Medical Officer of 4DMT.
  • “Current therapies do not adequately address Fabry-related cardiovascular manifestations, and cardiovascular disease is the most common cause of death in these patients.
  • “No approved therapy has been shown to definitively clear accumulated Gb3 from cardiomyocytes in patients with Fabry disease, which speaks to 4D-310’s highly differentiated profile.

Exactech Publishes New Machine Learning Research That Evaluates Fairness and Accuracy of AI Predictions

Retrieved on: 
Monday, November 27, 2023
Research, Technology, Medical Devices, Surgery, Other Technology, Electronic Design Automation, Health, Science, Artificial Intelligence, Other Science, Machine learning, Classification, Perception, SCB, Surgeon, Patient, Multimedia, Journal, Accuracy and precision, AI, MCID, Fairness, Medical device, Extremities, Shoulder

View the full release here: https://www.businesswire.com/news/home/20231127072917/en/

Key Points: 
  • View the full release here: https://www.businesswire.com/news/home/20231127072917/en/
    Exactech Publishes New Machine Learning Research That Evaluates Fairness and Accuracy of AI Predictions (Graphic: Business Wire)
    “This new machine learning study is important because it proposes a new statistical test methodology to evaluate the fairness of AI predictions,” said Chris Roche, Exactech’s Sr. Vice President, Extremities, “and because it establishes the clinically relevant criteria that determine acceptable differences in prediction accuracy between shoulder arthroplasty patients of different demographic status.
  • Comparing differences in accuracy between patient groups, the authors were able to identify precisely which patient groups received fair predictions and which patient groups received unfair predictions.
  • “Bias in AI clinical predictions can adversely impact decision making,” said Vikas Kumar, Exactech’s Vice President of Machine Learning.
  • As a result, it is critical to train and evaluate machine learning prediction tools against diverse datasets that are representative of all potential patients.

Mirum Pharmaceuticals Presents New Data at The Liver Meeting®

Retrieved on: 
Monday, November 13, 2023
Science, Biotechnology, Research, Pharmaceutical, General Health, Health, Infectious Diseases, Clinical Trials, Sleep, Hepatology, MDR3, Self-harm, Safety, Hepatocellular carcinoma, Bilirubin, Cholestasis, AASLD, Liver, Disease, Risk, Cholestatic pruritus, Hematology, Quality of life (healthcare), MYO5B, PFIC, Patient, Publication, Pregnancy, Nature, TJP2, Ascending cholangitis, GI, Hippeastrum mirum, Liver support system, Volixibat, ICP, IBAT, Progressive familial intrahepatic cholestasis, FIC, Augmented fifth, Growth, Doctor of Philosophy, Quality of life, AND, Itch, Medication, MCID, HIV disease progression rates, SBA, Chloride, Pharmaceutical industry, Nursing, Medical imaging, Dietary supplement, Boston, BSEP

Mirum Pharmaceuticals, Inc. (NASDAQ: MIRM) today announced new data from its LIVMARLI and volixibat programs presented at The Liver Meeting®, the American Association for the Study of Liver Diseases (AASLD) annual meeting in Boston, Massachusetts.

Key Points: 
  • Mirum Pharmaceuticals, Inc. (NASDAQ: MIRM) today announced new data from its LIVMARLI and volixibat programs presented at The Liver Meeting®, the American Association for the Study of Liver Diseases (AASLD) annual meeting in Boston, Massachusetts.
  • “PFIC is a chronic, debilitating cholestatic liver disease characterized by elevated bile acids and pruritus so severe that patients can be listed for liver transplant even in the absence of disease progression.
  • These data suggest overall improved liver health with maralixibat treatment in patients with PFIC that can be maintained long-term.
  • No clinically meaningful changes in liver enzyme levels or hematology parameters were observed after volixibat treatment.

Groundbreaking Study Finds Natural Formulation by MDbio – The Doctors BrandTM Significantly Improves Sleep, Anxiety, Stress, and Overall Well-being

Retrieved on: 
Tuesday, October 3, 2023
Volunteering, GABA, Theanine, Doctor of Pharmacy, Melatonin, University, Doctor, Anxiety, Sleep, Clinical trial, Public, Cannabinoid, Research, Harvard University, Maryland School, MCID, CBN, Harvard T.H. Chan School of Public Health, Public health, MD, Polypharmacology, University of Maryland School of Medicine, CBD, FACS, Faculty, Entrepreneurship, UCLA, THC, Pharmaceutical industry, Dietary supplement, Medicinal plants, Nutrient

Los Angeles, CA, Oct. 03, 2023 (GLOBE NEWSWIRE) -- In a momentous large-scale clinical trial, a MDSleep formulation by MDbio – The Doctors BrandTM was proven to significantly improve sleep compared to placebo.

Key Points: 
  • Los Angeles, CA, Oct. 03, 2023 (GLOBE NEWSWIRE) -- In a momentous large-scale clinical trial, a MDSleep formulation by MDbio – The Doctors BrandTM was proven to significantly improve sleep compared to placebo.
  • Results showed the formulation enhanced sleep onset, and supported restful and healthy sleep.
  • Furthermore, the product led to significant improvements in feelings of anxiety, stress, and well-being compared to placebo.
  • This study was one of history’s largest double-blind randomized placebo controlled trials to demonstrate effectiveness of a non-melatonin supplement for improving sleep.

aTyr Pharma Presents Efzofitimod Data Demonstrating Statistically Significant Improvements in Time to Relapse, FVC and Patient Reported Outcomes

Retrieved on: 
Monday, September 11, 2023
Sarcoidosis, Myelocyte, European Respiratory Society, International, University, Small RNA, Neuropilin, Scar, ILD, OCS, Toxicity, FVC, Inflammation, Scleroderma, ERS, MCID, Prednisone, Fibrosis, ATyr Pharma, Patient, SAN, Poster, Systemic scleroderma, International Congress on Tuberculosis, Safety, Pharmaceutical industry, Dietary supplement, PRO, Medicine

SAN DIEGO, Sept. 11, 2023 (GLOBE NEWSWIRE) -- aTyr Pharma, Inc. (Nasdaq: LIFE) (aTyr or the Company), a clinical stage biotechnology company engaged in the discovery and development of first-in-class medicines from its proprietary tRNA synthetase platform, today announced the results of a post-hoc analysis of data from its Phase 1b/2a study of efzofitimod in patients with pulmonary sarcoidosis. The analysis was presented in a poster at the European Respiratory Society (ERS) International Congress 2023, which is taking place September 9 – 13, 2023, in Milan, Italy. The poster is available on the Company’s website.

Key Points: 
  • 7.7% of patients in the 3.0 and 5.0 mg/kg efzofitimod group relapsed following steroid taper, compared to 54.4% in the placebo and efzofitimod 1.0 mg/kg group (p=0.017).
  • This is one of the few studies to demonstrate a steroid sparing effect of a drug associated with a significant improvement in patient outcome.
  • Dose dependent improvements in steroid burden, FVC and patient reported outcomes (PRO) were noted, though the study was not powered for efficacy.
  • These forms of ILD have limited therapeutic options and there is a need for safer and more effective, disease-modifying treatments that improve outcomes.

DIGITAL HEALTH PLATFORM IMPROVES ASTHMA AND DEPRESSION OUTCOMES IN TWO CLINICAL TRIALS

Retrieved on: 
Thursday, August 10, 2023
ACT, University College London, UCL, Asthma, Depression, Isabella Tree, University, RCT, Medicine, Harvard Medical School, Doctor of Philosophy, MCID, Juli, Literature, Odds ratio, Patient, Master of Science, Psychiatry, Digital, Pharmaceutical industry, Dermatology, MD

BOSTON, Aug. 10, 2023 /PRNewswire/ -- In two large randomized controlled trials (RCTs) conducted with patients diagnosed with two different chronic conditions, the digital health app juli demonstrated efficacy by improving outcomes in people with either asthma or depression compared to participants using an active control app, the company reported today.

Key Points: 
  • They were conducted in collaboration with University College London (UCL), ranked among the top 10 universities for life sciences and medicine globally.
  • juli joins a small group of digital health apps to have completed RCTs, considered the gold standard for the evaluation of medical interventions.
  • In both studies, the primary endpoint of statistically significant improvements compared to control was achieved, with p=0.020 in the asthma RCT and p=0.045 in the depression RCT.
  • "The fact that they've now demonstrated efficacy across two conditions as diverse as asthma and depression validates their approach and is very encouraging."

Nalu Medical, Inc. Presents Important New Clinical Data for PNS and SCS at ASPN 2023

Retrieved on: 
Thursday, July 20, 2023
Biotechnology, Health, Neurology, Clinical Trials, Human, Medicine, ODI, RCT, Spinal cord, Nalu, PNS, MCID, PGIC, Pain, Oswestry Disability Index, Patient, Physician, Engineering, Pain management, Spinal cord stimulator, ASPN, Society, Conference, PSP, SCS, Medical imaging, Neuroscience

The COMFORT RCT interim 6-month data show a 100% responder rate (≥50% reduction in pain) for subjects suffering from chronic peripheral neuralgia treated with the Nalu Peripheral Nerve Stimulation System (n=15).

Key Points: 
  • The COMFORT RCT interim 6-month data show a 100% responder rate (≥50% reduction in pain) for subjects suffering from chronic peripheral neuralgia treated with the Nalu Peripheral Nerve Stimulation System (n=15).
  • Additional highlights from the presentation include the following:
    78% mean pain relief from baseline.
  • The lead author, Dr. Mitchell Engle, noted, “Interim results show that the Nalu PNS System can provide sustained pain relief and functional improvement for patients with peripheral neuralgia.
  • This indicates that treatment with the Nalu SCS System provides a robust, holistic treatment response.” Nalu attributes results of this study to advanced engineering and the patented pulse stimulation pattern (PSP) waveform, which leverages multiple mechanisms of action in a single waveform3.

Digital Care Management Improves MSK Care, Force Therapeutics Report Shows

Retrieved on: 
Tuesday, July 18, 2023
Pros, CMS, ODI, Hip, Shoulder, Centers for Medicare & Medicaid Services, Disability, VBC, Center for Medicare and Medicaid Innovation, Element, ASES, MCID, Pain, Medicare, Hospital, Patient, Collection, Length of stay, KOOS, Force, MSK, BBC, Geisinger Health System, FFS, Therapy, SCB, Mental health, Knee, Medical device, Nursing, Pharmaceutical industry, Health insurance, Birth control

NEW YORK, July 18, 2023 /PRNewswire/ -- Force Therapeutics, the leading musculoskeletal (MSK) digital care management platform, announced today the release of a new report demonstrating that its virtual care management and remote engagement platform resulted in clinically significant improvements in patient-reported outcomes (PROs) including pain, function, mental health, and activities of daily living in MSK patients.

Key Points: 
  • Upcoming CMS incentives require providers to drive high-quality, value-based care through stronger patient engagement and care pathway compliance
    NEW YORK, July 18, 2023 /PRNewswire/ -- Force Therapeutics, the leading musculoskeletal (MSK) digital care management platform, announced today the release of a new report demonstrating that its virtual care management and remote engagement platform resulted in clinically significant improvements in patient-reported outcomes (PROs) including pain, function, mental health, and activities of daily living in MSK patients.
  • 90% of patients reported being "satisfied" or "very satisfied" with their surgical episodes combined with Force Therapeutics' care management plans.
  • "Providers must be prepared for the increasingly rigorous requirements surrounding care quality and outcomes, which necessitate the implementation of holistic digital and remote care solutions," said Bronwyn Spira, CEO and founder, Force Therapeutics.
  • "Without a comprehensive digital care strategy and strong patient engagement, providers will find it difficult to succeed in the rapidly evolving MSK care landscape," said Mark Lieberman, President and co-founder, Force Therapeutics.

Pliant Therapeutics Announces Positive Long-Term Data from the INTEGRIS-IPF Phase 2a Trial Demonstrating Bexotegrast was Well Tolerated at 320 mg with Durable Improvement Shown in FVC and Across Multiple Measures

Retrieved on: 
Sunday, April 30, 2023
University of Southern California, University, Week, Serum, HRCT, Telephone, Clinical trial, Pharmacokinetics, Vanderbilt University Medical Center, ILD, Keck School of Medicine of USC, VAS, Biomarker, QLF, Cough, MCID, Interest, Plasma, Fibrosis, Visual analogue scale, Conference call, SAE, Vital capacity, Walking, HIV disease progression rates, Patient, Standard of care, SAN, Webcast, Mortality, IPF, Therapy, Death, III, Conference, Idiopathic pulmonary fibrosis, Caregiver, Safety, Pharmaceutical industry, Medical imaging, Medicine, FVC

The 320 mg dose group met its primary and secondary endpoints demonstrating that bexotegrast was well tolerated up to 40 weeks and displayed a favorable pharmacokinetic profile.

Key Points: 
  • The 320 mg dose group met its primary and secondary endpoints demonstrating that bexotegrast was well tolerated up to 40 weeks and displayed a favorable pharmacokinetic profile.
  • The 320 mg group enrolled 21 patients in the active arm and 8 patients in the placebo arm.
  • The primary endpoint of the INTEGRIS-IPF trial was the evaluation of the safety and tolerability of bexotegrast.
  • Bexotegrast was well tolerated at 320 mg up to 40 weeks of treatment with no drug-related serious adverse events (SAE) reported.

DiscGenics Announces Positive Two-Year Clinical Data from Study of Discogenic Progenitor Cell Therapy for Degenerative Disc Disease

Retrieved on: 
Monday, January 23, 2023
Visual analogue scale, MD, Safety, University, NSAID, University of Tennessee Health Science Center, Ibuprofen, Low back pain, AACN Advanced Critical Care, Minimal important difference, Neurosurgery, Conference, North American Spine Society, Pain, Spine, VAS, IDCT, Cell, Aspirin, Trial of the century, FDA, Annual general meeting, MCID, American Academy, Synthetic control method, MRI, Philippine Orthopedic Center, NASS, Disability, ODI, Quality of life, Patient, SALT, DDD, Medical imaging, Birth control, Nursing, Pharmaceutical industry, Tissue, Intervertebral disc

SALT LAKE CITY, Jan. 23, 2023 /PRNewswire/ -- DiscGenics, Inc., a clinical stage biopharmaceutical company focused on developing regenerative cell-based therapies that alleviate pain and restore function in patients with degenerative diseases of the spine, today announced positive two-year clinical data from its first-in-human clinical study of IDCT (rebonuputemcel), an allogeneic discogenic progenitor cell therapy for lumbar degenerative disc disease (DDD).

Key Points: 
  • SALT LAKE CITY, Jan. 23, 2023 /PRNewswire/ -- DiscGenics, Inc. , a clinical stage biopharmaceutical company focused on developing regenerative cell-based therapies that alleviate pain and restore function in patients with degenerative diseases of the spine, today announced positive two-year clinical data from its first-in-human clinical study of IDCT (rebonuputemcel), an allogeneic discogenic progenitor cell therapy for lumbar degenerative disc disease (DDD).
  • In the low dose IDCT group (3,000,000 cell/mL; n=20), there was a trend in improvement of clinical outcomes, though inconsistent.
  • The 60-subject study was designed to evaluate the safety and preliminary efficacy of IDCT for the treatment of symptomatic lumbar degenerative disc disease versus vehicle and saline controls.
  • DiscGenics has submitted a full clinical study report to the U.S. Food & Drug Administration's (FDA) Office of Tissues and Advanced Therapies (OTAT).