scholarly journals A Phase 2b Clinical Trial Assessing Lomecel-B Infusion in Individuals with Aging Frailty: Study Design and Rationale

2021 ◽  
Vol 5 (Supplement_1) ◽  
pp. 672-672
Author(s):  
Kevin Ramdas ◽  
Ben Hitchinson ◽  
Lisa McClain-Moss ◽  
Keyvan Yousefi ◽  
Liliana Diaz ◽  
...  
Keyword(s):  
Physical Function ◽  
Multiple Organ ◽  
Geriatric Syndrome ◽  
Clinical Frailty Scale ◽  
Double Blind ◽  
Adverse Health Outcomes ◽  
Organ Systems ◽  
Patient Reported ◽  
Factor Α ◽  
And Function

Abstract Frailty is a common and important geriatric syndrome characterized by age-associated declines in physiology and function across multiple organ systems, which lead to increased vulnerability to adverse health outcomes. A biological mechanism that underlies the decline in physical function associated with aging frailty is chronic inflammation. The MSCs in Lomecel-B have immuno-modulatory capacity and control inflammation and the cytokine production of lymphocytes. An individual’s endogenous stem cell production decreases with age, this decrease likely contributes to reduced ability to regenerate and repair organs and tissues. Aging Frailty represents an exciting potential indication for cellular based therapies like Lomecel-B. This study is intended to evaluate the effects of Lomecel-B infusion compared to placebo on mobility and exercise tolerance, patient-reported physical function assessments and biomarkers for inflammation in individuals with Aging Frailty. This is a randomized, double-blind placebo-controlled, parallel multi-arm multicenter study enrolling adults aged 70-85 years identified as mildly or moderately frail per the CSHA Clinical Frailty Scale (CFS), with reduced six minute walk test (6MWT) and elevated Tumor Necrosis Factor-α (TNF-α), at screening. 150 subjects (30 per group) were randomized to receive a single peripheral intravenous infusion of 25, 50, 100, or 200 million doses, or placebo. Safety and efficacy assessments were conducted at 30, 90, 180, and 270 days after infusion. A follow up telephone call to subjects was placed at 365 days. We describe the design and rationale in detail of this 2b study assessing the effects of Lomecel-B on older adults with Aging Frailty.

scholarly journals Within-subjects Effects of Standardized Prosthetic Socket Modifications on Physical Function and Patient-Reported Outcomes

2021 ◽  
Author(s):  
William Anderst ◽  
Goeran Fiedler ◽  
Kentaro Onishi ◽  
Gina McKernan ◽  
Tom Gale ◽  
...  
Keyword(s):  
Physical Function ◽  
Patient Reported Outcomes ◽  
Design Method ◽  
Pressure Sensors ◽  
Residual Limb ◽  
Prosthetic Socket ◽  
Large Patient ◽  
Increased Risk ◽  
Patient Reported ◽  
And Function

Abstract • Background: Among the challenges of living with lower limb loss is the increased risk of long-term health problems that can be either attributed directly to the amputation surgery and/or prosthetic rehabilitation or indirectly to a disability-induced sedentary lifestyle. These problems are exacerbated by poorly fit prosthetic sockets. There is a knowledge gap regarding how the socket design affects in-socket mechanics, and how in-socket mechanics affect patient-reported comfort and function. The objectives of this study are: 1) to gain a better understanding of how in-socket mechanics of the residual limb in transfemoral amputees are related to patient-reported comfort and function, 2) to identify clinical tests that can streamline the socket design process, and 3) to evaluate the efficacy and cost of a novel, quantitatively informed socket optimization process.• Methods: Users of transfemoral prostheses will be asked to walk on a treadmill wearing their current socket plus 8 different check sockets with designed changes in different structural measurements that are likely to induce changes in residual limb motion, skin strain, and pressure distribution within the socket. Dynamic biplane radiography and pressure sensors will be used to measure in-socket residual limb mechanics. Patient-reported outcomes will also be collected after wearing each socket. The effects of in-socket mechanics on both physical function and patient-reported outcomes (aim 1) will be assessed using a generalized linear model. Partial correlation analysis will be used to examine the association between research grade measurements and readily available clinical measurements (aim 2). In order to compare the new quantitative design method to the Standard of Care, patient reported outcomes and cost will be compared between the two methods, utilizing the Wilcoxon Mann-Whitney non-parametric test (aim 3).• Discussion: Knowledge on how prosthetic socket modifications affect residual bone and skin biomechanics itself can be applied to devise future socket designs, and the methodology can be used to investigate and improve such designs, past and present. Apart from saving time and costs, this may result in better prosthetic socket fit for a large patient population, thus increasing their mobility, participation, and overall health-related quality of life. • Trial registration: clinicaltrials.gov: NCT05041998

scholarly journals A case of guillain-barré syndrome associated with post-covid infection during 2nd wave of coronavirus in india

2021 ◽  
pp. 342-344
Author(s):  
Anil Kumar Behera ◽  
Syed Naqueeb ul Hassan ◽  
Chaitanya Challa ◽  
K Divya Madhusudhan Reddy ◽  
V Bharat R Bhardwaj ◽  
...  
Keyword(s):  
Axonal Neuropathy ◽  
Inflammatory Cells ◽  
Multiple Organ ◽  
Guillain Barre Syndrome ◽  
Polymerase Chain Reaction Test ◽  
Immune Mediated ◽  
Organ Systems ◽  
Patient Reported ◽  
Guillain Barré Syndrome ◽  
Guillain Barré

The novel coronavirus-COVID-19 was detected in Wuhan city of Hubei Province in China on December 31, 2019. Coronaviruses are known to infect multiple organ systems, with respiratory complications being the most obvious symptoms. Although, neurological manifestations are quite rarely reported in cases of COVID-19. In this report, we present the case of a 57-year-old male patient reported with complaints of acute progressive symmetric quadriparesis and recently recovered from COVID-19. Two weeks prior to hospitalization, the patient suffered from cough and fever. The reverse transcriptase-polymerase chain reaction test for COVID-19 infection was positive. Electrodiagnostic tests showed that the patient had acute motor and sensory axonal neuropathy variant of Guillain-Barré Syndrome (GBS). COVID-19 virus stimulates the inflammatory cells and as a result, creates immune-mediated processes. GBS is an immune-mediated disorder. It is not clear whether COVID-19 infection induces the production of antibodies against specific gangliosides. Further investigations should be conducted in regards to the mechanism of GBS in patients with COVID-19 in the future.

scholarly journals Effect of Pain Education and Exercise on Pain and Function in Chronic Achilles Tendinopathy: Protocol for a Double-Blind, Placebo-Controlled Randomized Trial

10.2196/19111 ◽  
2020 ◽  
Vol 9 (11) ◽  
pp. e19111
Author(s):  
Andrew A Post ◽  
Ebonie K Rio ◽  
Kathleen A Sluka ◽  
G Lorimer Moseley ◽  
Emine O Bayman ◽  
...  
Keyword(s):  
Physical Function ◽  
Exercise Program ◽  
Achilles Tendinopathy ◽  
Double Blind ◽  
Pain Education ◽  
Double Blind Placebo ◽  
Biopsychosocial Approach ◽  
Primary Data Analysis ◽  
Patient Reported

Background Achilles tendinopathy (AT) rehabilitation traditionally includes progressive tendon loading exercises. Recent evidence suggests a biopsychosocial approach that incorporates patient education on psychosocial factors and mechanisms of pain can reduce pain and disability in individuals with chronic pain. This is yet to be examined in individuals with AT. Objective This study aims to compare the effects on movement-evoked pain and self-reported function of pain education as part of a biopsychosocial approach with pathoanatomical education for people with AT when combined with a progressive tendon loading exercise program. Methods A single-site, randomized, double-blind, placebo-controlled clinical trial will be conducted in a university-based hospital in a laboratory setting and/or by telehealth. A total of 66 participants with chronic (>3 months) midportion or insertional AT will be randomized for the Tendinopathy Education of the Achilles (TEAch) study. All participants will complete progressive Achilles tendon loading exercises over 12 weeks and will be encouraged to continue with self-selected exercises as tolerated. All participants will complete 6-7 one-to-one sessions with a physical therapist to progress exercises in a standardized manner over 8 weeks. During the last 4 weeks of the intervention, participants will be encouraged to maintain their home exercise program. Participants will be randomized to 1 of 2 types of education (pain education or pathoanatomic), in addition to exercise. Pain education will focus on the biological and psychological mechanisms of pain within a biopsychosocial framing of AT. Pathoanatomic education will focus on biological processes within a more traditional biomedical framework of AT. Evaluation sessions will be completed at baseline and 8-week follow-up, and self-reported outcome measures will be completed at the 12-week follow-up. Both groups will complete progressive Achilles loading exercises in 4 phases throughout the 12 weeks and will be encouraged to continue with self-selected exercises as tolerated. Primary outcomes are movement-evoked pain during heel raises and self-reported function (patient-reported outcome measure information system—Physical Function). Secondary outcomes assess central nervous system nociceptive processing, psychological factors, motor function, and feasibility. Results Institutional review board approval was obtained on April 15, 2019, and study funding began in July 2019. As of March 2020, we randomized 23 out of 66 participants. In September 2020, we screened 267 individuals, consented 68 participants, and randomized 51 participants. We anticipate completing the primary data analysis by March 2022. Conclusions The TEAch study will evaluate the utility of pain education for those with AT and the effects of improved patient knowledge on pain, physical function, and clinical outcomes. International Registered Report Identifier (IRRID) DERR1-10.2196/19111

scholarly journals Pain Assessment in Foot and Ankle Patients

2018 ◽  
Vol 3 (3) ◽  
pp. 2473011418S0006
Author(s):  
Meghan Kelly ◽  
David Bernstein ◽  
Ashlee MacDonald ◽  
John Ketz ◽  
Adolph Flemister ◽  
...  
Keyword(s):  
Pain Management ◽  
Physical Function ◽  
Patient Reported Outcomes ◽  
Pearson Correlation ◽  
Pain Level ◽  
Foot And Ankle ◽  
Moderate Correlation ◽  
Patient Reported ◽  
And Function

Category: Other Introduction/Purpose: The ability to accurately quantify a patient’s pain pre-operatively is advantageous in the preparation of post-operative expectations and pain management. The Numeric Pain Rating Scale (NPRS) is a popular method to identify patient pain level. Other patient reported outcomes are being collected, such as the Patient Reported Outcomes Measurement Information System (PROMIS) and has been suggested to be more accurate in measuring pain as well as physical function. The aim of this study was to 1) determine whether NPRS or PROMIS Pain Interference (PI) demonstrates a stronger association with physical function as determined by PROMIS Physical Function (PF) and 2) to determine which method better predicts post-surgical pain in a population of elective surgical foot and ankle patients. Methods: Prospective PROMIS PF, PI and NPRS (0-10) data was obtained for common foot and ankle elective surgical procedures (CPT codes 27698, 27870, 28285, 28289, 28300, 28705, 28730, 28750) from a multi-surgeon foot and ankle clinic between February 2015 until November 2017. Pearson correlation coefficients were used to determine the relationship between NPRS (0-10) and PROMIS domains (PI, PF) pre and post-operatively. Correlations were considered high (> 0.7), high moderate (0.6-0.69), moderate (0.4-0.6) or weak (<0.4). Results: A total of 502 patients found to have complete data sets and > 6 month follow up were evaluated (74% women, mean age 54+/- SD, mean follow-up 14.4 months, range 6-34 months). Pearson correlation evaluation of NPRS and PI revealed a moderate correlation in the pre- and postoperative setting. There was a high moderate negative correlation between PI and PF t-scores pre and postoperatively suggesting more pain and less function. However, the negative correlation between NPRS and PF pre- and postoperatively was weak indicating a poor relationship between NPRS pain assessment and function. There was a moderate correlation between pre- and postoperative scores in all domains of PROMIS while the correlation between pre- and postoperative NPRS scores was weak. Conclusion: In a population of elective surgical foot and ankle patients, the use of both NPRS and PROMIS can be utilized to assess pain level, however the PROMIS PI domain demonstrated a stronger relationship with PROMIS PF than NPRS. Furthermore, only the PROMIS domains demonstrated at least a moderate correlation between pre- and post-operative scores. PROMIS PI provides superior assessment of pre- and post-operative physical function and prediction of post-operative pain. PROMIS PI can be used to gauge a patient’s pre-operative level of pain and function and aid the surgeon in guiding post-operative patient expectations and pain management.

Patient-reported diarrhea impact on physical functioning and quality of life in clinical trial data submitted to the U.S. Food and Drug Administration.

2020 ◽  
Vol 38 (15_suppl) ◽  
pp. e19105-e19105
Author(s):  
Ting-Yu Chen ◽  
Bellinda King-Kallimanis ◽  
Lyna Merzoug ◽  
Mallorie Fiero ◽  
Jennifer J Gao ◽  
...  
Keyword(s):  
Breast Cancer ◽  
Quality Of Life ◽  
Clinical Trial Data ◽  
Life Questionnaire ◽  
Double Blind ◽  
Patient Reported ◽  
Related Quality ◽  
Cancer Trials ◽  
And Function

e19105 Background: Patient-reported outcomes can provide symptom and function data that complement standard oncology endpoints. Frequently, trials will conclude there was no clinically meaningful detriment to health-related quality of life (HRQL) or function, even when notable toxicity is observed. It is possible that mean change from baseline analyses obscures meaningful change in subgroups experiencing symptomatic toxicity. In this study, we explore how patients’ response to a diarrhea item related to physical function (PF) and HRQL in trials submitted to US FDA. Methods: We analyzed 3 randomized, double-blind breast cancer trials (early to late line metastatic) where diarrhea was a more common AE-symptom in the treatment arm, but there was not a large detriment in the mean change from baseline for HRQL and PF. Trials included the EORTC Quality of Life Questionnaire (QLQ-C30), which captures patient-reported HRQL, symptoms, and functioning. Higher scores (range 0-100) indicate better functioning and HRQL. Symptoms were measured with a 4-point scale; not at all to very much. Descriptive statistics were used to analyze diarrhea, PF, and HRQL over time. Results: Patients reporting very much diarrhea at month 3 had worse PF and HRQL compared to patients reporting no diarrhea . The range of difference between patients who reported very much diarrhea and those with none was 8-18 points for PF across trials. For HRQL scores, the range was 13–17 points worse. This trend was also seen in the control arm and at other times. Conclusions: In this set of breast cancer trials with differences in diarrhea by arm, reporting “no meaningful difference in PF or HRQL between the arms” is insufficient and potentially misleading. A more informative interpretation is that an exploratory analysis of HRQL and PF did not show in the investigational arm; there was a greater proportion of patients reporting diarrhea on the treatment arm; and patients reporting more frequent diarrhea reported lower HRQL and PF compared to patients with no diarrhea, regardless of arm. [Table: see text]

scholarly journals Counteracting age-related VEGF signaling insufficiency promotes healthy aging and extends life span

Science ◽  
2021 ◽  
Vol 373 (6554) ◽  
pp. eabc8479
Author(s):  
M. Grunewald ◽  
S. Kumar ◽  
H. Sharife ◽  
E. Volinsky ◽  
A. Gileles-Hillel ◽  
...  
Keyword(s):  
Life Span ◽  
Healthy Aging ◽  
Vascular Diseases ◽  
Tumor Burden ◽  
Multiple Organ ◽  
Vegf Signaling ◽  
Established Risk Factor ◽  
Age Related ◽  
Organ Systems ◽  
And Function

Aging is an established risk factor for vascular diseases, but vascular aging itself may contribute to the progressive deterioration of organ function. Here, we show in aged mice that vascular endothelial growth factor (VEGF) signaling insufficiency, which is caused by increased production of decoy receptors, may drive physiological aging across multiple organ systems. Increasing VEGF signaling prevented age-associated capillary loss, improved organ perfusion and function, and extended life span. Healthier aging was evidenced by favorable metabolism and body composition and amelioration of aging-associated pathologies including hepatic steatosis, sarcopenia, osteoporosis, “inflammaging” (age-related multiorgan chronic inflammation), and increased tumor burden. These results indicate that VEGF signaling insufficiency affects organ aging in mice and suggest that modulating this pathway may result in increased mammalian life span and improved overall health.

scholarly journals Challenges in defining Long COVID: Striking differences across literature, Electronic Health Records, and patient-reported information

2021 ◽  
Author(s):  
Halie M. Rando ◽  
Tellen D. Bennett ◽  
James Brian Byrd ◽  
Carolyn Bramante ◽  
Tiffany J. Callahan ◽  
...  
Keyword(s):  
Electronic Health Records ◽  
Multiple Organ ◽  
Health Records ◽  
Health Crisis ◽  
Organ Systems ◽  
Wide Range ◽  
Patient Reported ◽  
Electronic Health ◽  
Novel Coronavirus

Since late 2019, the novel coronavirus SARS-CoV-2 has introduced a wide array of health challenges globally. In addition to a complex acute presentation that can affect multiple organ systems, increasing evidence points to long-term sequelae being common and impactful. As the worldwide scientific community forges ahead with efforts to characterize a wide range of outcomes associated with SARS-CoV-2 infection, the proliferation of available data has made it clear that formal definitions are needed in order to design robust and consistent studies of Long COVID that consistently capture variation in long-term outcomes. In the present study, we investigate the definitions used in the literature published to date and compare them against data available from electronic health records and patient-reported information collected via surveys. Long COVID holds the potential to produce a second public health crisis on the heels of the pandemic. Proactive efforts to identify the characteristics of this heterogeneous condition are imperative for a rigorous scientific effort to investigate and mitigate this threat.

Responder analysis of patient-reported outcomes measurement information system (PROMIS) physical function (PF) and worst stiffness among patients with tenosynovial giant cell tumors (TGCT) in the ENLIVEN study.

2019 ◽  
Vol 37 (15_suppl) ◽  
pp. e18236-e18236
Author(s):  
William D. Tap ◽  
Rebecca M Speck ◽  
Xin Ye ◽  
Emanuela Palmerini ◽  
Silvia Stacchiotti ◽  
...  
Keyword(s):  
Physical Function ◽  
Rating Scale ◽  
Numerical Rating Scale ◽  
Cumulative Distribution ◽  
Measurement Information ◽  
Global Rating ◽  
Giant Cell Tumors ◽  
Double Blind ◽  
Meaningful Improvement ◽  
Patient Reported

e18236 Background: The double-blind, randomized, placebo-controlled phase 3 ENLIVEN study in TGCT demonstrated a significant tumor response at week 25 by RECIST for pexidartinib (39% vs. 0% for placebo) and improvement in joint function and symptoms. The aim of this analysis was to identify a threshold score for responder definitions for PROMIS-PF scale and Worst Stiffness Numerical Rating Scale (WS-NRS) and compared responder rates for pexidartinib versus placebo. Methods: Anchor- and distribution-based estimates were calculated, and cumulative distribution function (CDF) plots were generated to derive responder definition threshold estimates. Anchor- and distribution-based results and CDFs were evaluated through triangulation, following FDA PRO Guidance, to determine a single responder definition threshold (i.e., meaningful change) for each instrument. The proportion of responders at Week 25 between treatments was compared with Fisher’s Exact Test (2-sided). Results: 120 patients were randomized to pexidartinib (n = 61) and placebo (n = 59) and assessed through 25 weeks of treatment. Anchor-based analysis showed one-level improvement on the patient global rating of PF item was associated with a mean change of 4.0 on PROMIS-PF. Distribution-based estimates (0.5 SD and 1 SEM) for PROMIS-PF were 2.8 and 2.5, respectively. For WS-NRS, a response of “A little improved” by patients on the perception of stiffness item was associated with a mean change of 1.1. The distribution-based estimates for the WS-NRS item were 0.9 and 0.5, respectively. This resulted in the following responder definition thresholds: ≥3 points for PROMIS-PF and ≥1 for WS-NRS improvement. A greater proportion of pexidartinib as compared to placebo patients were responders by PROMIS-PF (30% vs. 5%, p < 0.001) and WS-NRS (39% vs. 19%, p = 0.02) at week 25, respectively. Conclusions: Triangulation yielded responder definitions of ≥3 points for PROMIS-PF and ≥1 for WS-NRS. With these definitions, a greater proportion of patients treated with pexidartinib compared to placebo had meaningful improvement in physical function and stiffness.

scholarly journals Effect of Pain Education and Exercise on Pain and Function in Chronic Achilles Tendinopathy: Protocol for a Double-Blind, Placebo-Controlled Randomized Trial (Preprint)

2020 ◽  
Author(s):  
Andrew A Post ◽  
Ebonie K Rio ◽  
Kathleen A Sluka ◽  
G Lorimer Moseley ◽  
Emine O Bayman ◽  
...  
Keyword(s):  
Physical Function ◽  
Exercise Program ◽  
Achilles Tendinopathy ◽  
Double Blind ◽  
Pain Education ◽  
Double Blind Placebo ◽  
Biopsychosocial Approach ◽  
Primary Data Analysis ◽  
Patient Reported

BACKGROUND Achilles tendinopathy (AT) rehabilitation traditionally includes progressive tendon loading exercises. Recent evidence suggests a biopsychosocial approach that incorporates patient education on psychosocial factors and mechanisms of pain can reduce pain and disability in individuals with chronic pain. This is yet to be examined in individuals with AT. OBJECTIVE This study aims to compare the effects on movement-evoked pain and self-reported function of pain education as part of a biopsychosocial approach with pathoanatomical education for people with AT when combined with a progressive tendon loading exercise program. METHODS A single-site, randomized, double-blind, placebo-controlled clinical trial will be conducted in a university-based hospital in a laboratory setting and/or by telehealth. A total of 66 participants with chronic (&gt;3 months) midportion or insertional AT will be randomized for the Tendinopathy Education of the Achilles (TEAch) study. All participants will complete progressive Achilles tendon loading exercises over 12 weeks and will be encouraged to continue with self-selected exercises as tolerated. All participants will complete 6-7 one-to-one sessions with a physical therapist to progress exercises in a standardized manner over 8 weeks. During the last 4 weeks of the intervention, participants will be encouraged to maintain their home exercise program. Participants will be randomized to 1 of 2 types of education (pain education or pathoanatomic), in addition to exercise. Pain education will focus on the biological and psychological mechanisms of pain within a biopsychosocial framing of AT. Pathoanatomic education will focus on biological processes within a more traditional biomedical framework of AT. Evaluation sessions will be completed at baseline and 8-week follow-up, and self-reported outcome measures will be completed at the 12-week follow-up. Both groups will complete progressive Achilles loading exercises in 4 phases throughout the 12 weeks and will be encouraged to continue with self-selected exercises as tolerated. Primary outcomes are movement-evoked pain during heel raises and self-reported function (patient-reported outcome measure information system—Physical Function). Secondary outcomes assess central nervous system nociceptive processing, psychological factors, motor function, and feasibility. RESULTS Institutional review board approval was obtained on April 15, 2019, and study funding began in July 2019. As of March 2020, we randomized 23 out of 66 participants. In September 2020, we screened 267 individuals, consented 68 participants, and randomized 51 participants. We anticipate completing the primary data analysis by March 2022. CONCLUSIONS The TEAch study will evaluate the utility of pain education for those with AT and the effects of improved patient knowledge on pain, physical function, and clinical outcomes. INTERNATIONAL REGISTERED REPORT DERR1-10.2196/19111

scholarly journals Bezafibrate Improves Mitochondrial Fission and Function in DNM1L Deficient Patient Cells

2019 ◽  
Author(s):  
Liza Douiev ◽  
Ruth Sheffer ◽  
Gabriella Horvath ◽  
Ann Saada
Keyword(s):  
De Novo ◽  
Neurodevelopmental Disorder ◽  
Mitochondrial Fission ◽  
Multiple Organ ◽  
Mitochondrial Morphology ◽  
Main Role ◽  
Atp Production ◽  
Organ Systems ◽  
Mitochondrial Functions ◽  
And Function

Mitochondria are involved in many cellular processes and their main role is cellular energy production. They constantly undergo fission and fusion, and these counteracting processes are under strict balance. The cytosolic dynamin-related protein 1, Drp1 or dynamin-1-like protein (DNM1L) mediates mitochondrial and peroxisomal division. Defects in the DNM1L gene results in a complex neurodevelopmental disorder with heterogeneous symptoms affecting multiple organ systems. Currently there is no curative treatment available for this condition. We have previously described a patient with a de novo heterozygous c.1084G&gt;A (p.G362S) DNM1L mutation and studied the effects of a small molecule, Bezafibrate, on mitochondrial functions in this patient&rsquo;s fibroblasts compared to controls. Bezafibrate normalized growth on glucose-free medium, ATP production, oxygen consumption and s improved mitochondrial morphology in patient&rsquo;s fibroblasts, albeit concomitantly causing a mild increase ROS production. Further studies would be needed to show the consistency of the response to Bezafibrate, possibly using fibroblasts from patients with different mutations in DNM1L, and this treatment should be confirmed in clinical trials. However, taking into account the favorable effects in our study, we suggest that Bezafibrate could be a possible treatment option for patients with certain DNM1L mutations.

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