Introduction: Palovarotene is a retinoic acid receptor gamma agonist that was studied in phase-2 and phase-3 clinical trials for the inhibition of new heterotopic ossification (HO) in fibrodysplasia ossificans progressiva (FOP). Despite numerous setbacks and regulatory delays, palovarotene is now the first approved FOP treatment in the U.S.A. Canada and Australia but remains unapproved in Europe where concerns surrounding the drug and its path to regional market authorization persist.
Areas Covered: The developmental history of palovarotene and an overview of the clinical trials and the regulatory approval journey are discussed by global FOP experts.
Expert Opinion: While post hoc analyses indicate that palovarotene may have modest benefits for the inhibition of new HO formation in FOP, a number of limitations and concerns remain about its generalized use. Although the long-term risks and benefits of treatment with palovarotene remain unknown, the regional approval of palovarotene marks a milestone for the FOP community at the very beginning of a new era of clinical trials.
Download full-text PDF |
Source |
|---|---|
| http://dx.doi.org/10.1080/14656566.2025.2452938 | DOI Listing |
Publication Analysis
Top Keywords
Similar Publications
Safety and immunogenicity of an mRNA-1273 vaccine booster in adolescents.
Hum Vaccin Immunother
December 2025
Research and Development, Infectious Disease, Moderna, Inc., Cambridge, MA, USA.
Safety, immunogenicity, and effectiveness of an mRNA-1273 50-μg booster were evaluated in adolescents (12-17 years), with and without pre-booster SARS-CoV-2 infection. Participants who had received the 2-dose mRNA-1273 100-µg primary series in the TeenCOVE trial (NCT04649151) were offered the mRNA-1273 50-μg booster. Primary objectives included safety and inference of effectiveness by establishing noninferiority of neutralizing antibody (nAb) responses after the booster compared with the nAb post-primary series of mRNA-1273 among young adults in COVE (NCT04470427).
View Article and Find Full Text PDFBackground: Age-related macular degeneration (AMD), a condition of multifactorial origin, is a major cause of irreversible vision loss in industrialized countries. The dry late stage of the disease, known as geographic atrophy (GA), is characterized by progressive loss of photoreceptor cells and retinal pigment epithelial cells in the central retina. An estimated 300 000 to 550 000 people in Germany suffer from GA.
View Article and Find Full Text PDFMindfulness Training vs Recovery Support for Opioid Use, Craving, and Anxiety During Buprenorphine Treatment: A Randomized Clinical Trial.
JAMA Netw Open
January 2025
Department of Psychiatry, Harvard Medical School, Boston, Massachusetts.
Importance: During buprenorphine treatment for opioid use disorder (OUD), risk factors for opioid relapse or treatment dropout include comorbid substance use disorder, anxiety, or residual opioid craving. There is a need for a well-powered trial to evaluate virtually delivered groups, including both mindfulness and evidence-based approaches, to address these comorbidities during buprenorphine treatment.
Objective: To compare the effects of the Mindful Recovery Opioid Use Disorder Care Continuum (M-ROCC) vs active control among adults receiving buprenorphine for OUD.
The Statistical Fragility of Functional Outcomes for Arthroscopic Rotator Cuff Repair With and Without Acromioplasty: A Systematic Review and Meta-analysis.
Am J Sports Med
January 2025
Department of Orthopaedic Surgery and Rehabilitation, University of Texas Medical Branch, Galveston, Texas, USA.
Background: Views surrounding acromioplasty at the time of arthroscopic rotator cuff repair (RCR) have shifted dramatically over time. In recent years, various studies have argued against acromioplasty, citing equivocal functional outcomes after arthroscopic RCR with or without acromioplasty.
Purpose: To assess the statistical fragility of functional outcomes after arthroscopic RCR with and without acromioplasty using the reverse continuous fragility index (RCFI).
Prehabilitation for Chilean frail elderly people - pre-surgical conditioning protocol - to reduce the length of stay: randomized control trial.
Minerva Anestesiol
December 2024
Department of Anesthesiology, School of Medicine, Pontificia Universidad Católica de Chile, Santiago, Chile.
Background: Frail elderly patients have a higher risk of postoperative morbidity and mortality. Prehabilitation is a potential intervention for optimizing postoperative outcomes in frail patients. We studied the impact of a prehabilitation program on length of stay (LOS) in frail elderly patients undergoing elective surgery.
View Article and Find Full Text PDFWant AI Summaries of new PubMed Abstracts delivered to your In-box?
Enter search terms and have AI summaries delivered each week - change queries or unsubscribe any time!