Observational study

Shockwave Reducer Demonstrates Consistent, Positive Results in ‘Real-World’ Study

Retrieved on: 
Monday, April 8, 2024

These findings confirm the positive, ‘real-world’ outcomes of Shockwave Reducer, a novel technology for the treatment of refractory angina.

Key Points: 
  • These findings confirm the positive, ‘real-world’ outcomes of Shockwave Reducer, a novel technology for the treatment of refractory angina.
  • Results were presented earlier today at the 73rd Annual Scientific Sessions of the American College of Cardiology in Atlanta, GA.
    REDUCER-I is a post-market, multi-center, observational study designed to collect long-term outcomes on Shockwave Reducer in a large, ‘real-world’ population of 400 patients.
  • “Results from the REDUCER-I study continue to demonstrate the safety and effectiveness of Shockwave Coronary Sinus Reducer as a novel therapy for refractory angina,” said Dr. Verheye.
  • Results from COSIRA-II are intended to support the regulatory filing for US FDA approval of Shockwave Reducer.

Inozyme Pharma Announces Positive Topline Data from Ongoing Phase 1/2 Trials of INZ-701 in Adults with ABCC6 Deficiency (PXE) and ENPP1 Deficiency

Retrieved on: 
Monday, April 8, 2024

BOSTON, April 08, 2024 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc. (Nasdaq: INZY) (“the Company” or “Inozyme”), a clinical-stage rare disease biopharmaceutical company developing novel therapeutics for the treatment of pathologic mineralization and intimal proliferation, today announced positive topline safety, pharmacokinetic (PK), pharmacodynamic (PD) and exploratory efficacy data from the Company’s ongoing Phase 1/2 clinical trials of INZ-701 in adults with ABCC6 Deficiency (PXE, pseudoxanthoma elasticum) and ENPP1 Deficiency.

Key Points: 
  • “We are excited by the excellent safety and preliminary efficacy profile of INZ-701 in adults with ABCC6 Deficiency,” said Douglas A. Treco, Ph.D., CEO of Inozyme Pharma.
  • The patients were assigned to three dose cohorts of INZ-701: 0.2 mg/kg (n=3), 0.6 mg/kg (n=3), and 1.8 mg/kg (n=4).
  • For trial design details, please see the section entitled “INZ-701 in ABCC6 Deficiency Phase 1/2 Clinical Trial Design” below.
  • For trial design details, please see the section entitled “INZ-701 in ENPP1 Deficiency Phase 1/2 Clinical Trial Design” below.

Pair of Digital Health Research Papers Coauthored by Analysis Group Demonstrate Promises of Wearables to Generate Reliable Site-Less Patient-Reported Outcomes in Rheumatoid Arthritis

Retrieved on: 
Tuesday, April 9, 2024

This technology enables clinicians to develop more sensitive and patient-centric endpoints in RA clinical trials and real-world studies.

Key Points: 
  • This technology enables clinicians to develop more sensitive and patient-centric endpoints in RA clinical trials and real-world studies.
  • "Digital health technologies and machine learning augment patient reported outcomes to remotely characterise rheumatoid arthritis," published in February by Nature's npj Digital Medicine (Creagh, A.P., et al.
  • "Digital health technologies and machine learning augment patient reported outcomes to remotely characterise rheumatoid arthritis."
  • "Patient-centric assessment of rheumatoid arthritis using a smartwatch and bespoke mobile app in a clinical setting."

Lost for words? Research shows art therapy brings benefits for mental health

Retrieved on: 
Tuesday, April 9, 2024

Art therapy uses creative processes, primarily visual art such as painting, drawing or sculpture, with a view to improving physical health and emotional wellbeing.

Key Points: 
  • Art therapy uses creative processes, primarily visual art such as painting, drawing or sculpture, with a view to improving physical health and emotional wellbeing.
  • Art therapy can be used to support treatment for a wide range of physical and mental health conditions.
  • In a study published this week in the Journal of Mental Health, we found art therapy was associated with positive outcomes for children and adolescents in a hospital-based mental health unit.

An option for those who can’t find the words

  • Where possible, after finishing an artwork, a person can explore the meaning of their work with the art therapist, translating unspoken symbolic material into verbal reflection.
  • However, as the talking component is less central to the therapeutic process, art therapy is an accessible option for people who may not be able to find the words to describe their experiences.
  • Art therapy has also been linked to improved outcomes for people with a range of physical health conditions.

Our research

  • While most care takes place in the community, a small proportion of young people require care in hospital to ensure their safety.
  • However, these “restrictive practices” are associated with negative effects such as post-traumatic stress for patients and health professionals.
  • Read more:
    'An arts engagement that's changed their life': the magic of arts and health

    Our research looked at more than six years of data from a child and adolescent mental health hospital ward in Australia.

  • Confirming the effect of a therapeutic intervention requires controlled clinical trials where people are randomly assigned one treatment or another.
  • Although ours was an observational study, randomised controlled trials support the benefits of art therapy in youth mental health services.

What do young people think?

  • In previous research we found art therapy was considered by adolescents in hospital-based mental health care to be the most helpful group therapy intervention compared to other talk-based therapy groups and creative activities.
  • In research not yet published, we’re speaking with young people to better understand their experiences of art therapy, and why it might reduce distress.

A promising area


The burgeoning research showing the benefits of art therapy for both physical and especially mental health highlights the value of creative and innovative approaches to treatment in health care. There are opportunities to expand art therapy services in a range of health-care settings. Doing so would enable greater access to art therapy for people with a variety of physical and mental health conditions.

  • She has received funding from the Health Education and Training Institute (HETI) for the Mental Health Research Award.
  • Iain Perkes works for the University of New South Wales and the Sydney Children's Hospitals Network which is part of NSW Health.
  • He is affiliated with Neuroscience Research Australia (NeuRA) and the Health Education and Training Institute (HETI, NSW Health).

Design Therapeutics Outlines Progress Across GeneTAC™ Platform and Announces Fourth Quarter and Full Year 2023 Financial Results

Retrieved on: 
Tuesday, March 19, 2024

R&D Expenses: Research and development (R&D) expenses were $11.0 million for the quarter ended December 31, 2023, and $57.1 million for the year ended December 31, 2023.

Key Points: 
  • R&D Expenses: Research and development (R&D) expenses were $11.0 million for the quarter ended December 31, 2023, and $57.1 million for the year ended December 31, 2023.
  • G&A Expenses: General and administrative (G&A) expenses were $4.1 million for the quarter ended December 31, 2023, and $21.1 million for the year ended December 31, 2023.
  • Net Loss: Net loss was $11.8 million for the quarter ended December 31, 2023, and $66.9 million for the year ended December 31, 2023.
  • Cash Position and Operating Runway: Cash, cash equivalents and marketable securities were $281.8 million as of December 31, 2023.

JenaValve’s Trilogy THV System Highlighted at CRT 2024

Retrieved on: 
Tuesday, March 19, 2024

IRVINE, Calif., March 19, 2024 (GLOBE NEWSWIRE) -- JenaValve Technology, Inc., developer and manufacturer of the Trilogy™ Transcatheter Heart Valve (THV) System, today announced highlights from the Cardiovascular Research Technologies (CRT) Conference 2024.

Key Points: 
  • IRVINE, Calif., March 19, 2024 (GLOBE NEWSWIRE) -- JenaValve Technology, Inc., developer and manufacturer of the Trilogy™ Transcatheter Heart Valve (THV) System, today announced highlights from the Cardiovascular Research Technologies (CRT) Conference 2024.
  • JenaValve’s Trilogy THV system, a TAVR system designed to treat patients with symptomatic, severe aortic regurgitation (ssAR), and symptomatic, severe aortic stenosis (ssAS), was featured in multiple scientific sessions highlighting the significant unmet need for devices to treat ssAR.
  • This session reviewed an observational study examining outcomes of patients with ssAR treated with off-label THV’s versus a device designed to treat ssAR.
  • Results showed significantly better technical and device success in the Trilogy arm, with significantly less valve embolization, need for a second valve, and post-procedural aortic regurgitation.

Otsuka Works with Verily to Launch Longitudinal Mental Health Registry to Advance Novel Research in Psychiatry

Retrieved on: 
Thursday, April 4, 2024

Otsuka Pharmaceutical Development & Commercialization, Inc. (Otsuka), a global pharmaceutical company, announces the launch of My Mental Health Journey , a longitudinal registry study that aims to advance research in depression and mental health.

Key Points: 
  • Otsuka Pharmaceutical Development & Commercialization, Inc. (Otsuka), a global pharmaceutical company, announces the launch of My Mental Health Journey , a longitudinal registry study that aims to advance research in depression and mental health.
  • Verily Life Sciences LLC (Verily), a precision health technology company, will recruit participants and run the study.
  • View the full release here: https://www.businesswire.com/news/home/20240404726198/en/
    The My Mental Health Journey registry will enroll U.S.-based adults experiencing symptoms of depression to help Otsuka better understand mental health conditions, such as major depressive disorder, across time and in the real-world setting.
  • Participants in the My Mental Health Journey may receive compensation for study activities, educational resources, updates on future clinical studies, and other opportunities to help advance depression research.

Koneksa Announces Merck Joins Data Syndication Partnership Program Around Parkinson’s Digital Biomarkers Observational Study

Retrieved on: 
Wednesday, March 20, 2024

Koneksa , a healthcare technology company pioneering evidence-based digital biomarkers, announced that Merck, known as MSD outside of the United States and Canada, has joined its Data Syndication Partnership program for the LEARNS observational study, which seeks to develop digital biomarkers in neurodegenerative disorders from smart phone-based assessments and wearable technologies.

Key Points: 
  • Koneksa , a healthcare technology company pioneering evidence-based digital biomarkers, announced that Merck, known as MSD outside of the United States and Canada, has joined its Data Syndication Partnership program for the LEARNS observational study, which seeks to develop digital biomarkers in neurodegenerative disorders from smart phone-based assessments and wearable technologies.
  • As a member, Merck researchers will have real-time access to data and results from the ongoing study.
  • The high-precision longitudinal data and in-clinic data from the LEARNS study will guide future development of digital biomarkers of disease progression in Parkinson’s disease.
  • Data syndication gives partners immediate and continuous access to data and results as the study progresses, and access to the study dashboard from Koneksa’s proprietary platform for real-time study monitoring, enabling syndication partners to learn from the study as it progresses.

New Research Reveals Genomic Profile of Seborrheic Dermatitis and Answers Key Questions on Immune Response and Skin Barrier Dysfunction

Retrieved on: 
Saturday, March 9, 2024

“We are excited to have successfully employed noninvasive tape-stripping techniques developed from studying these diseases to study an understudied and undertreated inflammatory skin disease, seborrheic dermatitis.

Key Points: 
  • “We are excited to have successfully employed noninvasive tape-stripping techniques developed from studying these diseases to study an understudied and undertreated inflammatory skin disease, seborrheic dermatitis.
  • These findings will help establish the groundwork for greater understanding of this very common condition.”
    “The pathophysiology of seborrheic dermatitis has been poorly understood.
  • In addition, the skin barrier disruption observed in seborrheic dermatitis has unique molecular underpinnings, primarily in the tight junction of the epithelial skin cells and lipid metabolism pathways.
  • These data demonstrate that seborrheic dermatitis is an immune disease with a uniquely polarized profile distinct from atopic dermatitis and plaque psoriasis.

BioCryst Presents New Real-world Data Showing Rapid, Substantial and Sustained HAE Attack Rate Reductions After Beginning ORLADEYO® (berotralstat) Treatment

Retrieved on: 
Friday, February 23, 2024

“These additional analyses of real-world use of ORLADEYO show that any person living with HAE has the potential to experience a rapid, substantial and sustained reduction in their monthly attack rate with ORLADEYO.

Key Points: 
  • “These additional analyses of real-world use of ORLADEYO show that any person living with HAE has the potential to experience a rapid, substantial and sustained reduction in their monthly attack rate with ORLADEYO.
  • These analyses present the overall attack rate progression and attack rate progression stratified by severity (i.e., number of attacks at baseline), prior prophylaxis and C1-INH level and function.
  • These findings further demonstrate that ORLADEYO can help maintain disease control in patients with lower baseline attack rates and further reduce attack rates in patients with more active disease.
  • Berotralstat Prophylaxis Reduces HAE Attack Rates Regardless of Baseline Attacks: Real-World Outcomes; Poster #012; Friday, February 23, 3:15-4:15 p.m.