deflazacort / Generic mfg. - LARVOL DELTA

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

Circinate balanitis in HLA B27 spondyloarthrithis: A report of two cases. (PubMed, Int J STD AIDS) - "He was on treatment with tofacitinib, deflazacort, and sulfasalazine. The final diagnosis was circinate balanitis with primary ankylosing spondylitis. Both cases highlight the diagnostic challenge of distinguishing between different forms of spondyloarthritis in the presence ofcircinate balanitis."

Journal • Ankylosing Spondylitis • Conjunctivitis • Dermatology • Dermatopathology • Immunology • Inflammatory Arthritis • Ocular Infections • Ocular Inflammation • Ophthalmology • Psoriasis • Rheumatology • Seronegative Spondyloarthropathies • Spondylarthritis

Conservative Management of Acute Myocarditis and Thrombotic Microangiopathy after Elevidys (Delandistrogene Moxeparvovec) (ASGCT 2025) - "Case: He remained ambulatory with current DMD medications of deflazacort 36 mg daily and givinostat (held 2 weeks prior to GT)...Baseline cardiac medications were enalapril, eplerenone, and carvedilol...The day prior, he started 60 mg daily of prednisone for immunosuppression per the label...The next day (Day-4), he reported palpitations while telemetry showed atrial fibrillation, which resolved after 20 minutes of esmolol infusion...Complement blockade with eculizumab was considered for rAAV-associated TMA, but our multispecialty gene therapy team consisting of neurology, cardiology, hematology, and nephrology recommended a conservative watchful waiting approaching... TMA is a poorly understood rAAV toxicity. Complement activation is hypothesized to be a central component of its pathophysiology. However, the scope of rAAV drugs that incite TMA as well as its optimal management remains to be defined."

Late-breaking abstract • Atrial Fibrillation • Cardiovascular • Fibrosis • Gene Therapies • Heart Failure • Hematological Disorders • Hypertension • Inflammation • Muscular Dystrophy • Myositis • Nephrology • Renal Disease • Thrombocytopenia • CST3

Administrative healthcare data to identify and describe patients with rare diseases: the case of Duchenne muscular dystrophy. (PubMed, Recenti Prog Med) - "This study of administrative data has identified patients potentially affected by DMD, a rare disease, from a large sample of INHS beneficiaries, and assessed their healthcare pathway. This is useful for regulatory purposes and for improved access to emerging innovative therapies."

Journal • Observational data • Retrospective data • Cardiomyopathy • Cardiovascular • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy • Musculoskeletal Diseases • Orthopedics • Rare Diseases • Respiratory Diseases

Management practices in Italy on adrenal insufficiency in young people with Duchenne muscular dystrophy on steroid treatment (ESPE-ESE 2025) - "The most commonly used GC regimen was daily deflazacort (86%), followed by daily prednisone (26%), intermittent deflazacort (14%), and intermittent prednisone (3%). To our knowledge, this is the first survey addressing AI management practices among physicians caring for individuals with DMD in Italy. Most physicians involved in DMD care manage fewer than 50 patients. There are notable differences in approach for minor, moderate, and major stress, with nearly a quarter of respondents not prescribing parenteral hydrocortisone in major stress situations."

Anesthesia • Duchenne Muscular Dystrophy • Endocrine Disorders • Genetic Disorders • Muscular Dystrophy • Musculoskeletal Diseases • Musculoskeletal Pain • Nephrology • Orthopedics • Pain • Pediatrics • Renal Disease

Exercise combined with corticoid/omega-3 therapy positively affected skeletal and cardiac muscles in middle aged mdx mice. (PubMed, Int J Exp Pathol) - "Exercised mdx received deflazacort (1.2 mg/kg; gavage) alone or combined with omega-3 (300 mg/kg; gavage)...The muscles studied showed a reduction in inflammation biomarkers (NF-κB, IL-6 and TNF-α) and fibrosis (TGF-β and fibronectin) and an increase in calsequestrin (calcium buffering protein) and PGC1-α (muscle integrity marker). In conclusion, exercise alone or associated with corticosteroid/omega-3 therapy benefited limb, diaphragm and cardiac dystrophic muscles in middle-aged mdx mice."

Journal • Preclinical • Duchenne Muscular Dystrophy • Fibrosis • Genetic Disorders • Immunology • Inflammation • Muscular Dystrophy • IL6 • TGFB1 • TNFA

Zydus Lifesciences gets USFDA nod for Jaythari (Deflazacort) tablets (CNBC-TV18) - "Zydus Lifesciences Ltd...on Friday, April 11, announced that it has received final approval from the United States Food and Drug Administration (USFDA) for its Jaythari (Deflazacort) Tablets. The approved strengths include 6 mg, 18 mg, 30 mg, and 36 mg, enabling Zydus to launch a generic version of Emflaza Tablets in the US market...Jaythari is indicated for the treatment of Duchenne muscular dystrophy (DMD)—a rare genetic disorder that causes progressive muscle weakness and degeneration."

ANDA • Duchenne Muscular Dystrophy

Avascular Necrosis of the Hip Joint from Unjustified SteroidUse: A Case Series Report (IARS-SOCCA 2025) - "He had taken 30 mg of Deflazacort daily for a year during and after a COVID-19 infection...Steroid use was stopped, and a treatment plan including NSAIDs, physiotherapy, and lifestyle changes was initiated.Case 4: A 20-year-old electrician from Vikrampur presented on January 25, 2023, with six months of right hip pain after consuming Megestrol syrup for weight gain...Avascular necrosis (AVN) can progress to subchondral fractures and joint incongruity, often requiring arthroplasty. Judicious steroid use under medical supervision, strict adherence to guidelines, and curbing OTC steroid sales are crucial. Transparency in herbal ingredients and regional research is essential to mitigate risks."

Clinical • Asthma • Back Pain • Immunology • Infectious Disease • Musculoskeletal Diseases • Musculoskeletal Pain • Novel Coronavirus Disease • Orthopedics • Pain • Respiratory Diseases

Deflazacort Vs. Prednisolone in Acute-stage ABPA (clinicaltrials.gov) - P2/3 | N=150 | Active, not recruiting | Sponsor: Post Graduate Institute of Medical Education and Research, Chandigarh | Trial completion date: Dec 2024 ➔ Jul 2025 | Trial primary completion date: Dec 2024 ➔ Jul 2025

Trial completion date • Trial primary completion date • Allergic Bronchopulmonary Aspergillosis • Pulmonary Disease • Respiratory Diseases

Becker Muscular Dystrophy and Nephrotic-Range Proteinuria: Chance or True Association? (PubMed, Br J Hosp Med (Lond)) - "Results The patient accepted oral deflazacort for two months...Chronic kidney impairment in pediatric BMD warrants attention, and the role of kidney biopsy in characterizing DMD/BMD-associated nephropathy should be explored. Given the lifelong and heterogeneous nature of BMD, multicenter study and long-term follow-up are required to better understand the progression, underlying mechanisms, and clinical outcomes of renal complications in these patients."

Clinical • Journal • Becker Muscular Dystrophy • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy • Nephrology • Pediatrics • Renal Disease

Observational study of changes to glucocorticosteroid prescribing patterns in duchenne muscular dystrophy in the UK over the last decade. (PubMed, J Neurol Neurosurg Psychiatry) - "The use of deflazacort and daily regimes has steadily increased across the UK North Star Network in the last decade. This study provides one of the largest up-to-date real-world set of data of evolution in prescription patterns and the occurrence of side-effects in different groups of GC-treated DMD."

Journal • Observational data • CNS Disorders • Duchenne Muscular Dystrophy • Gastrointestinal Disorder • Genetic Disorders • Insomnia • Muscular Dystrophy • Sleep Disorder