deflazacort / Generic mfg. - LARVOL DELTA

STEROID-RESPONSIVE NEPHROTIC SYNDROME IN TWIN SISTERS (ISN-WCN 2026) - "Treatment was started with deflazacort 60 mg/m2/day for 4 weeks followed by tapering and slow reduction...In genetic studies targeted for podocyte molecules, such as, NPHS1, NPHS2, PLCE1, ACTN4, CD2AP, WT-1, TRPC6, LAMB2, were all negative.Conclusion We reports a pair of identical female twins who contemporaneously developed idiopathic SSNS. We found that they showed familial feature, however, genetic defects of podocyte molecules were not detected yet."

Glomerulonephritis • Nephrology • Renal Disease • CD2AP • HLA-DRB1 • LAMB2 • LAMC1 • NPHS1 • TRPC6 • WT1

Results of a phase II open-label, multiple-dose study of vamorolone (VBP15-006) in 7- to < 18-year-old boys with duchenne muscular dystrophy. (PubMed, J Neurol) - P, P2 | "Vamorolone demonstrated a consistent safety profile in 7- to < 18-year-old boys with DMD. Switching to 6 mg/kg/day vamorolone appeared to mitigate adrenal insufficiency risk. There was no negative effect on growth, and catch-up growth occurred in previously CS-treated individuals switching to vamorolone."

Journal • P2 data • Duchenne Muscular Dystrophy • Endocrine Disorders • Genetic Disorders • Muscular Dystrophy • Nephrology • Renal Disease

Management of Duchenne Muscular Dystrophy in Clinical Practice: A Survey-Based Study in Spain. (PubMed, Neurol Ther) - "These findings reveal a proactive clinical landscape characterized by early diagnosis and high adherence to international pharmacological standards. However, persistent gaps in multidisciplinary access and patient-centered monitoring highlight critical unmet needs. The implementation of a dedicated multidisciplinary framework and standardized management protocols is essential for optimizing the care of patients with DMD."

Journal • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy • Pediatrics

Low-Dose Deflazacort Reduces Postoperative Pain and Inflammation Following Primary Total Knee Arthroplasty: A Randomized Controlled Trial. (PubMed, J ISAKOS) - "Low-dose oral deflazacort following primary TKA was associated with modest but consistent improvements in pain, function, sleep quality, and inflammatory markers, with an excellent safety profile. Although clinical improvements did not exceed minimal clinically important differences, the findings suggest that low-dose deflazacort may serve as a safe adjunct in multimodal postoperative recovery protocols with potential implications for reducing opioid consumption and improving patient satisfaction."

Journal • CNS Disorders • Immunology • Inflammation • Orthopedics • Osteoarthritis • Pain • Rheumatology • Sleep Disorder • CRP • IL6

State of art of oral corticosteroids in children with acute asthma and wheezing. (PubMed, Ital J Pediatr) - No abstract available

Journal • Review • Asthma • Immunology • Pulmonary Disease • Respiratory Diseases

Melkersson-Rosenthal syndrome with facial swelling and palsy and associated diagnostic challenges: A case report. (PubMed, Med Int (Lond)) - "The patient responded well to a 6-month regimen of deflazacort, epinastine and methotrexate. Treatment focuses on symptom control with corticosteroids; immunomodulators or biologics may be used in refractory cases. Early recognition is essential, even in the absence of the full triad."

Journal • CNS Disorders • Dermatology • Hemophilia

A Rare Co-occurrence of Duchenne Muscular Dystrophy and Glycerol Kinase Deficiency Associated With Xp21 Contiguous Gene Deletion Syndrome: A Case Report. (PubMed, Cureus) - "He maintains deflazacort therapy and follows a nutrition plan based on a fat-restricted diet and avoidance of prolonged fasting to prevent further metabolic crises...In our case, it was possible to diagnose complex kinase deficiency along with Duchenne muscular dystrophy. Consequently, it was optimal for multi-profile medical care accompanied by an adequate nutritional plan."

Journal • Developmental Disorders • Duchenne Muscular Dystrophy • Dyslipidemia • Genetic Disorders • Hypertriglyceridemia • Hypoglycemia • Mental Retardation • Metabolic Disorders • Muscular Dystrophy • GK

Refractory Gingival Enlargement: A Critical Oral Clue to Early-Stage Granulomatosis With Polyangiitis-A Case Report and Literature Review. (PubMed, Case Rep Dent) - "Multidisciplinary management was initiated with systemic immunosuppression (oral azathioprine and deflazacort) and topical therapy, alongside optimization of the patient's comorbid conditions. The presence of "strawberry gingivitis" serves as a crucial, pathognomonic clinical sign. We propose a diagnostic and management protocol for oral GPA to improve patient outcomes."

Journal • Dental Disorders • Diabetes • Endocrine Disorders • Inflammation • Metabolic Disorders • Pain • Rare Diseases • Vasculitis

Endocrine and metabolic complications in a national cohort of Slovene children and adolescents with Duchenne muscular dystrophy: real-world criteria for transition to vamorolone therapy. (PubMed, Front Endocrinol (Lausanne)) - "Two patients were corticosteroid-naive, the rest treated with deflazacort (average treatment duration 5.4, range 1.1-11.3 yrs). We propose that ambulatory, non-corticosteroid naive patients with pathological fractures, markedly reduced bone density, short stature, or significant growth deceleration could be considered for the therapeutic transition. Accordingly, we established national clinical criteria to guide individualized therapeutic transitions, aimed at optimizing clinical outcomes and ensuring efficient allocation of healthcare resources."

Journal • Real-world evidence • Diabetes • Duchenne Muscular Dystrophy • Genetic Disorders • Metabolic Disorders • Muscular Dystrophy • Musculoskeletal Diseases • Obesity • Orthopedics • Pediatrics • Type 2 Diabetes Mellitus

Corticosteroid treatment in Duchenne muscular dystrophy. (PubMed, Arch Pediatr) - "Corticoids are started and maintained at the theoretical dose of 0.75 mg/kg/day of prednisone/prednisolone or 0.9 mg/kg/day of deflazacort, adjusting the dose to weight if the benefit-risk ratio favors treatment. The only reasons to discontinue treatment when the patient and family are adhering to it are the uncontrolled side effects. Thus, in many countries including France, as of late 2025, the only routinely prescribed symptomatic treatment for DMD remains conventional corticosteroids, which will be discussed in this article, followed by a description of vamorolone, a dissociative steroidal compound, and givinostat, a histone deacetylase inhibitor."

Journal • Review • Cardiomyopathy • Cardiovascular • Duchenne Muscular Dystrophy • Genetic Disorders • Infectious Disease • Muscular Dystrophy • Obesity • Osteoporosis • Respiratory Diseases • Rheumatology

Paper-Based Reaction Devices as Accelerated Platforms in Forced Degradation Studies. (PubMed, J Pharm Sci) - "Strips of cellulose paper were employed as reactive microenvironments to investigate the degradation of two model drugs, cyclobenzaprine (CBP) and deflazacort (DFL), under oxidative, acidic, basic, and metal-ion stress conditions. The PRD approach is proposed as an investigational and preformulation-level tool for early-phase stability assessment, complementing rather than replacing conventional stress testing methods. Its simple, robust, and high-throughput format offers a valuable alternative for rapid stability evaluation during the early stages of drug development and formulation design."

Journal

Recognition of Diffuse Pustules and Hemorrhagic Crusts as Early Warning Signs of Invasive Candidiasis in a Patient With Toxic Epidermal Necrolysis. (PubMed, Clin Case Rep) - "Herein, we report a 41-year-old female known case of IgA nephropathy admitted for having TEN after a recent intake of Deflazacort, developed systemic candidiasis during admission, and was successfully treated with oral fluconazole. Early detection of diffuse pustular and hemorrhagic crusted lesions with immediate antifungal treatment in TEN is crucial to prevent systemic complications and enhance patient outcomes."

Journal • Candidiasis • Glomerulonephritis • Hematological Disorders • IgA Nephropathy • Renal Disease • Steven-Johnson Syndrome

Hand X-ray metrics for assessing bone health and fracture risk in Duchenne muscular dystrophy: a cross-sectional study. (PubMed, Eur J Pediatr) - "Hand X-ray-derived metrics, as BHI and BA, represent a non-invasive tool for bone health assessment in DMD. BHI may serve as a supportive biomarker when DXA is limited, and BA may carry potential as a fracture risk indicator, although prospective validation is warranted."

Journal • Observational data • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy • Musculoskeletal Diseases • Orthopedics

Predicting Treatment Effect on the Loss of Ability to Rise From Floor in Duchenne Muscular Dystrophy Using Individual Trajectories: An Application of Joint Modeling in FOR-DMD (ISPOR-EU 2025) - P3 | "The FOR-DMD trial (NCT01603407) included 196 boys with DMD aged 4-7, randomized to daily prednisone, daily deflazacort, or intermittent prednisone. A joint model, developed using the FOR-DMD dataset, provides a robust framework for predicting treatment effects on the LoR in DMD patients, utilizing individual TTR trajectories and other covariates. This approach can predict the effect of new treatments on LoR based on observed TTR trajectories."

Clinical • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

Evaluating in-vitro cellular toxicity through the analysis of nitrite ions variation at printed electrochemical strips. (PubMed, Talanta) - "Beyond detection, the sensor was successfully employed to monitor the anti-inflammatory response to three mechanistically distinct drugs (erucin, naproxen, and deflazacort), demonstrating its suitability for probing cellular toxicity in different systems. Combining portability, low cost, and operational simplicity, this platform provides a practical alternative to conventional assays for quickly evaluating the therapeutic efficacy in in-vitro models."

Journal • Preclinical • Cardiovascular • Oncology

Joint Modelling of Growth and Motor Function Centiles in Corticosteroids Treated Boys With Duchenne Muscular Dystrophy. (PubMed, Eur J Neurol) - "This work describes the complex relationships between motor function, CS treatment and growth and provides insights for conversations about the relative benefits and negative effects of CS."

Journal • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

Targeting Cellular Senescence in Dystrophin-/-/Utrophin-/-Double Knockout Mice Improves Musculoskeletal Health and Increases Lifespan. (PubMed, Pharmacol Res) - "Co-administration of ruxolitinib and deflazacort also synergistically ameliorated skeletal muscle and heart pathology. Therefore, targeting senescent cells with ruxolitinib represents a promising approach for treating DMD patients but warrants further studies in humans."

Journal • Preclinical • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy • Musculoskeletal Diseases • CD68 • CDKN1A • MIF

PNEUMOCYSTIS PNEUMONIA IN DUCHENNE MUSCULAR DYSTROPHY: A CASE REPORT OF AN UNDERRECOGNIZED COMPLICATION (CHEST 2025) - "Glucocorticoids, particularly deflazacort, are a cornerstone of DMD therapy, helping to reduce muscle inflammation, improve muscle strength and ambulation, reduce scoliosis, delay pulmonary function loss and cardiomyopathy onset, and increase survival.1 Generally, daily doses equivalent to prednisone 20 mg or higher is a known risk factor for pneumocystis pneumonia (PJP), and prophylaxis using TMP-SMX is recommended.2 Although individual management varies, glucocorticoid therapy in DMD is often started at a prednisone equivalent of 0.75 mg/kg daily and titrated up to 30-40 mg if functional decline continues.3 However, PJP risk and prophylaxis recommendations in DMD is rarely if ever documented. We report a case of PJP in a DMD patient on long term glucocorticoids, a mainstay treatment in DMD. This case highlights the underrecognized risk of PJP in the DMD population and the need for investigation and discussion of PJP prophylaxis in DMD patients on chronic higher dose..."

Case report • Clinical • Cardiomyopathy • Cardiovascular • Cough • Duchenne Muscular Dystrophy • Genetic Disorders • Infectious Disease • Inflammation • Muscular Dystrophy • Pneumonia • Respiratory Diseases

Efficacy and safety of deflazacort in diabetic subjects infected with SARS-CoV-2. (PubMed, Multidiscip Respir Med) - "Deflazacort demonstrated a lower incidence of hospital admission for glycaemic decompensation compared to standard treatment with dexamethasone/methylprednisolone in SARS-CoV-2 positive outpatients. There were no differences in COVID-19-related hospitalizations between the two groups."

Journal • Diabetes • Infectious Disease • Metabolic Disorders • Novel Coronavirus Disease • Respiratory Diseases

Short term daily deflazacort decreases membrane permeability and increases maximum force in mdx mice. (PubMed, Am J Physiol Cell Physiol) - "Using ex vivo lumbrical muscle preparations, we found that levels of inter-contraction calcium accumulation significantly correlated with force decline during repeated isometric contractions in all deflazacort-treated mice, and a trend for lower aberrant calcium accumulation was seen following 3 weeks of treatment. Given that some protective effects were reduced or not present in a preclinical model of DMD with longer term steroid treatment, these data provide important evidence for beneficial use of short-term deflazacort."

Journal • Preclinical • Duchenne Muscular Dystrophy • Genetic Disorders • Inflammation • Muscular Dystrophy

Height, weight, and body mass index trajectories and their correlation with functional outcome assessments in boys with Duchenne muscular dystrophy. (PubMed, Dev Med Child Neurol) - "Changes in anthropometric measures after glucocorticoid initiation are associated with COA performance and larger correlations closer to the age of loss of ambulation. This emphasizes the need for weight management strategies and discussions that support treatment."

Clinical • Journal • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

The remarkable effects of the ionized medical water Asea® in 3 boys with Duchenne dystrophy: Three case reports. (PubMed, World J Methodol) - "ARS has significant indirect antioxidant effects via NRF2 and deserves extensive trials in children with DMD, as an adjuvant to corticoids or as a substitute in DMD patients who refuse corticoids. Future trials should also focus on ARS as an adjuvant in many types of acute/chronic infectious/non-infectious diseases where cellular oxidative stress is involved."

Journal • Developmental Disorders • Duchenne Muscular Dystrophy • Genetic Disorders • Mental Retardation • Muscular Dystrophy • MB

Patient demographics, clinical characteristics and genetic mutations of DMD and BMD patients in Qatar Epidemiological and genetic profile of Duchenne muscular dystrophy and Becker muscular dystrophy patients in Qatar: a retrospective cohort study. (PubMed, Front Pediatr) - "Corticosteroids were prescribed in 14 patients (38%), most commonly deflazacort and prednisone. Our cohort reveals earlier diagnosis but lower life expectancy compared to international standards, likely due to lower corticosteroid and cardioprotective use. The findings support the need for strengthened multidisciplinary and early genetic-based interventions in Qatar."

Journal • Retrospective data • ADHD (Impulsive Aggression) • Alzheimer's Disease • Attention Deficit Hyperactivity Disorder • Autism Spectrum Disorder • Becker Muscular Dystrophy • CNS Disorders • Cognitive Disorders • Duchenne Muscular Dystrophy • Epilepsy • Gene Therapies • Genetic Disorders • Muscular Dystrophy • Orthopedics • Pediatrics • Psychiatry • Respiratory Diseases

A Rare Case of Lichen Planus/Discoid Lupus Erythematosus Overlap Syndrome and Its Successful Treatment. (PubMed, Clin Case Rep) - "Subsequently, the patient was prescribed 200 mg of azathioprine daily, along with the corticosteroid Deflazacort. Notable improvement in the lesions was observed after 4-5 weeks of follow-up."

Journal • Dermatology • Dermatopathology • Discoid Lupus Erythematosus • Immunology • Inflammatory Arthritis • Lichen Planus • Lupus • Pain

Predicting trajectories of the north star ambulatory assessment total score in Duchenne muscular dystrophy. (PubMed, PLoS One) - "Among N = 416 patients at baseline, mean age was 8.2 years, mean NSAA total score was 24, and 61% were receiving prednisone and 39% deflazacort, with the majority having been treated with daily corticosteroid regimens (69%) relative to other regimens (31%). In conclusion, trajectories of ambulatory motor function in DMD, as measured by the NSAA total score, can be well-predicted using readily available baseline characteristics. We discuss applications of these predictions to DMD drug development."

Journal • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy