deflazacort / Generic mfg. - LARVOL DELTA

Long-term survival in adults with Duchenne muscular dystrophy treated with corticosteroids (ERS 2026) - "15 patients were prescribed systemic corticosteroids (1 deflazacort, 1 hydrocortisone, 13 prednisolone). Corticosteroids were stopped in 9 patients during follow-up. The overall survival was shorter for those who continued taking corticosteroids (22.3±3.1 years, n=6) compared to those who stopped them (25.2±4.8 years, n=9) or were never prescribed (28.3±2.4 years, n=6, p=0.01)."

Clinical • CNS Disorders • Diabetes • Duchenne Muscular Dystrophy • Endocrine Disorders • Genetic Disorders • Infectious Disease • Inflammation • Metabolic Disorders • Muscular Dystrophy • Musculoskeletal Diseases • Nephrology • Orthopedics • Osteoporosis • Renal Disease • Respiratory Diseases

Balancing Benefits and Risks: Long-term Use of Daily versus Intermittent Corticosteroids in Duchenne Muscular Dystrophy. (PubMed, J Neurol Neurosurg Psychiatry) - "Daily CS resulted in significantly slower decline in motor function at the expense of reduced height and increased fracture risk. These findings from real-world long-term data reveal that differences between treatment regimens increase over time, highlighting the critical role of such data in guiding individualised shared decision-making."

Journal • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy • Musculoskeletal Diseases • Orthopedics • Pediatrics

CHARACTERIZATION OF PATIENTS WITH IMMUNE THROMBOCYTOPENIA IN SPAIN. PRELIMINARY RESULTS FROM RESTI: SPANISH REGISTRY OF PRIMARY IMMUNE THROMBOCYTOPENIA (ISTH 2026) - "Corticosteroids were the most frequently used first-line therapy (95%), with prednisone (67.3%) and dexamethasone (39.7%) being the most commonly prescribed agents. Methylprednisolone (13.2%) and deflazacort (0.9%) were used less frequently...Second-line and subsequent therapies were widely used, particularly thrombopoietin receptor agonists, including eltrombopag (45.4%), avatrombopag (37.9%), and romiplostim (28.8%). Fostamatinib was prescribed in 12.1% of patients, and rituximab in 10%...Ultimately, RESTI aims to support harmonization of clinical practice and to serve as a reference framework for future national and international collaborative studies. Table or Figure Upload (1) Page 3 Table or Figure Upload (2) DOI*10.1016/j.rpth.2026.104420"

Clinical • Hematological Disorders • Immune Thrombocytopenic Purpura • Immunology • Thrombocytopenia • Thrombocytopenic Purpura

OMENUTS: Omega-3-Based Nutraceutical Supplementation as Adjunctive Therapy in Adults With Otitis Media With Effusion: A Multicenter, Double-Blind, Randomized Placebo-Controlled Trial (clinicaltrials.gov) - P4 | N=501 | Completed | Sponsor: Isola Tiberina - Gemelli Isola Hospital, Rome, Italy

New P4 trial • Otorhinolaryngology

Disproportionality analysis of sex-stratified adverse event signals in growth impairment: Insights from the FDA adverse event reporting system. (PubMed, Medicine (Baltimore)) - "Deflazacort showed a male-exclusive signal (ROR 58.25, 95% CI 41.27-82.21), while imatinib displayed a higher risk in females (ROR 15.55, 95% CI 11.29-21.42) compared to males (ROR 4.17, 95% CI 2.91-5.99). These findings generate the hypothesis that sex-disaggregated pharmacovigilance in pediatrics, particularly for growth-modulating therapies, may reveal differential reporting patterns. Should these signals be validated in controlled prospective studies, they could inform the development of customized monitoring frameworks and dosing strategies aimed at potentially mitigating risks in hypothesized high-risk subgroups."

Adverse events • Journal • Pediatrics

Pharmacodynamic Response of Circulating Proteins to Different Corticosteroid Types and Regimens in Duchenne Muscular Dystrophy (ICNMD 2026) - P3 | "Moreover, most newer therapies are used in combination with steroids (prednisone, deflazacort, and vamorolone). We studied the dose-response of serum proteins between intermittent and daily prednisone, and differential response to daily prednisone vs deflazacort in a large-scale, untargeted fashion. We validated some serum proteins previously reported as differentially responsive to prednisone vs deflazacort, but also identified newer differentially responsive biomarkers. Our work may help to better understand the mechanism of action of steroids, the predictive potential of these biomarkers and their importance to accelerated regulatory pathways, help refine therapies, define synergistic interactions between novel treatments and steroids, and potentially use as surrogate/secondary outcomes in trials."

PK/PD data • Duchenne Muscular Dystrophy • Gene Therapies • Genetic Disorders • Muscular Dystrophy

Post-marketing safety surveillance and signal characterization of the novel dissociative steroid Vamorolone in Duchenne muscular dystrophy: a comparative disproportionality analysis based on FAERS data. (PubMed, Front Pharmacol) - "To comprehensively evaluate the real-world post-marketing safety profile of the novel dissociative steroid Vamorolone and perform a comparative analysis against the traditional glucocorticoid deflazacort. Nevertheless, known risks and unexpected hypothesis-generating signals-such as notable HPA axis suppression and early-onset psychiatric disturbances-remain critical safety concerns. Clinicians should interpret these differential reporting patterns with caution and rigorously monitor neurobehavioral and adrenal health during the first three months, with further external validation warranted."

Journal • P4 data • CNS Disorders • Duchenne Muscular Dystrophy • Genetic Disorders • Infectious Disease • Mental Retardation • Muscular Dystrophy • Musculoskeletal Diseases • Orthopedics • Osteoporosis • Psychiatry • Rheumatology • Urinary Incontinence

Patient Characteristics and Use of Vamorolone in Adult Patients With DMD in Germany and Austria (ICNMD 2026) - "Six patients were corticosteroid (CS) naïve, 16 had prior prednisone and three prior deflazacort treatment. This first real-world analysis demonstrates clinical efficacy of vamorolone regarding stabilization or even mild improvement in adult DMD patients who were CS-naïve, had discontinued previous CS treatment, or switched from other CS. This data further highlights the importance of continued corticosteroid treatment beyond loss of ambulation."

Clinical • Cushing’s Disease • Duchenne Muscular Dystrophy • Endocrine Disorders • Genetic Disorders • Muscular Dystrophy • Obesity • Osteoporosis

Genetic Modifier Associations With Steroid Safety Outcomes in Duchenne Muscular Dystrophy (ICNMD 2026) - "Traditional corticosteroids like prednisone and deflazacort can result in weight gain, adrenal suppression, growth stunting, and reduced bone biomarkers, while vamorolone, a newly approved dissociative steroid, has shown an improved safety profile, especially on bone health. Al though based on a small sample size and needing additional studies for confirmation, we provide initial evidence of the association of genetic modifiers at an early age with common steroid safety concerns including for weight gain, which is a common reason for steroids prescribed at sub-recommended doses. These findings may be important for clinical trials and care."

Clinical • CNS Disorders • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy • CDX2

Prescription Patterns in Rheumatoid Arthritis Patients at a Tertiary Care Hospital: A Prospective Cross-Sectional Study. (PubMed, Cureus) - "Methotrexate was the predominant DMARD (75.8%), while diclofenac (20.0%) and deflazacort (18.9%) were the most frequently used NSAID and corticosteroid, respectively.  RA predominantly affects middle‑aged females with moderate disease duration and a notable comorbidity burden. Treatment patterns show strong reliance on conventional DMARDs, particularly methotrexate, with adjunct use of NSAIDs and corticosteroids, while biologics remain underused."

Journal • Observational data • Cardiovascular • Diabetes • Hematological Disorders • Hypertension • Immunology • Inflammatory Arthritis • Metabolic Disorders • Rheumatoid Arthritis • Rheumatology

Multimodal Assessment of Bone Health and Functional Status in Adults With Duchenne Muscular Dystrophy (ICNMD 2026) - "The majority of patients (78.4%) were on chronic corticosteroid therapy (86.2%% deflazacort and 13.8% prednisone) and 16.2% stopped treatment prior to the study due to side effects. Adults with DMD exhibit severe functional impairment, reduced muscle strength, and a high skeletal burden. The high fracture rate and low Z-scores highlight a critical need for improved and earlier bone health diagnosis and optimized management. Advanced 3D imaging (PCCT) may be a feasible alternative to DXA and may provide essential insight into fracture risk in this population."

Clinical • CNS Disorders • Cough • Depression • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy • Musculoskeletal Diseases • Orthopedics • Osteoporosis • Respiratory Diseases

The DMD-NEEDS Study: Real-World Management of Duchenne Muscular Dystrophy in Spain. (ICNMD 2026) - "Corticosteroid therapy, predominantly deflazacort, was prescribed by 95.1% of neurologists and typically initiated at ages 4-5 (66.7%)... The DMD-NEEDS study reveals an encouraging landscape in Spain, marked by high adherence to best practices like early diagnosis and widespread corticosteroid use. While these findings affirm positive trends, they also highlight critical areas for national optimization, particularly improving access to multidisciplinary care and mandating the routine application of validated HRQoL assessment tools to ensure holistic, patient-centered care."

Clinical • Real-world • Real-world evidence • CNS Disorders • Duchenne Muscular Dystrophy • Gene Therapies • Genetic Disorders • Muscular Dystrophy

SMAD6 Heterozygous Loss-of-Function Variant as a Potential Modifier of Duchenne Muscular Dytrosphy (DMD) (ICNMD 2026) - "Daily deflazacort was initiated at 4 years of age and continuously maintained, with dosage adjusted according to body weight... This case supports the concept that additional genetic factors may modulate DMD severity. SMAD6 partial loss-of-function emerges as a plausible disease modifier and therapeutic target, although its pleiotropic role—particularly in cardiovascular development—warrants caution. Further studies are needed to validate its modifier effect in preclinical DMD models."

Cardiovascular • CNS Disorders • Developmental Disorders • Duchenne Muscular Dystrophy • Fibrosis • Gastrointestinal Disorder • Genetic Disorders • Immunology • Muscular Dystrophy • LMNA • SETD5 • TGFB1

Disease progression and economic burden of duchenne muscular dystrophy: A retrospective study using Swedish register data. (PubMed, J Neuromuscul Dis) - "Glucocorticoid use was high (84% on either prednisolone or deflazacort)...DMD imposes a significant economic burden in Sweden, with direct non-medical costs far exceeding direct medical costs. These findings highlight the need for and value of early and effective interventions to delay disease progression and alleviate societal burden."

HEOR • Journal • Retrospective data • CNS Disorders • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

Meta-analysis of clinical trials assessing the safety of pharmacological treatments for muscular degeneration in Duchenne muscular dystrophy. (PubMed, BMC Pharmacol Toxicol) - "Precise ADR estimates can support decision-making in DMD care, helping clinicians balance safety and efficacy, address family concerns, and promote adherence by contextualizing treatment risks."

Journal • Retrospective data • Dermatology • Duchenne Muscular Dystrophy • Genetic Disorders • Hematological Disorders • Muscular Dystrophy

Vamorolone for Duchenne Muscular Dystrophy: A Cross-Trial Efficacy Comparison With Classic Corticosteroids From the FOR-DMD Trial. (PubMed, Neurology) - P2b, P3 | "Vamorolone demonstrated numerically similar TTSTAND velocity changes to prednisone and deflazacort at 1 year; however, interpretations of differences are limited by 95% CIs crossing minimally important difference thresholds. Further evidence of the growth-protective effect of vamorolone was observed; however, all treatments increased BMI. Vamorolone provides a linear growth-protective classic corticosteroid alternative."

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

Effects of Sex and Fed State Differences on the Pharmacokinetics of Drugs in Humans. (PubMed, Drug Res (Stuttg)) - "This study evaluates the impact of sex and food intake on key pharmacokinetics parameters-maximum plasma concentration, time to maximum plasma concentration, area under the curve to last time point, area under the curve extrapolated to infinity, and elimination half-life-for amlodipine, dapagliflozin, empagliflozin, rivaroxaban, deferiprone, deflazacort, and siponimod. Sex and fed state significantly influence the pharmacokinetics of several drugs. These findings underscore the necessity of considering these factors in clinical dosing regimens and drug development to optimize therapeutic outcomes."

Journal • PK/PD data

Elimination Profile of Deflazacort and Metabolites in Urine and Plasma Following Oral Administration: Relevance to Sports Drug Testing. (PubMed, Drug Test Anal) - "Cortisol levels were suppressed post-dose, indicating systemic GC activity, but recovered by 72 h in most subjects. The findings support the use of DES as the primary urinary marker for DEF detection, confirm the suitability of the MRL of 30 ng/mL and support the recommendation of a 3-day washout period due to interindividual variability."

Journal

Acute Pancreatitis as a Therapeutic Complication in Case of Paroxysmal Nocturnal Hemoglobinuria. (PubMed, Ann Afr Med) - "A 32-year-old female with PNH developed acute pancreatitis 10 months after initiation of danazol and deflazacort therapy. Healthcare providers managing PNH in African settings must remain vigilant for drug-induced pancreatitis when using traditional therapies. Enhanced monitoring protocols and improved access to safer complement inhibitors are essential for optimizing patient outcomes while minimizing treatment-related complications in resource-limited environments."

Journal • Complement-mediated Rare Disorders • Hematological Disorders • Inflammation • Pancreatitis • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases • Renal Disease

Delphy study on epidemiology, clinical management, disease burden, and treatment in paediatric patients with Duchenne muscular dystrophy in Spain. (PubMed, Neurologia (Engl Ed)) - "The study reports updated data on the epidemiology and clinical management of DMD in Spain. It also highlights the importance of early diagnosis, multidisciplinary management and the need for a new therapy that improves the safety profile of traditional glucocorticoids to provide an improved quality of life for patients with DMD."

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

Symmetrical flexural and intertriginous rash related to corticosteroids (PubMed, Rev Alerg Mex) - "We emphasize the importance of studying these types of patients and seeking alternatives to corticosteroids for future prescription, given their widespread use in medicine."

Journal • Acute Respiratory Distress Syndrome • Dermatitis • Dermatology • Immunology • Rare Diseases • Urticaria

Successful Management of a Chronic Pedal Ulcer Secondary to Snake Bite: A Case Report. (PubMed, Cureus) - "The patient was started on a course of anti-inflammatory therapy with deflazacort and colchicine, alongside standard wound care and pain control. This case highlights that the key to managing such longstanding, refractory ulcers lies in recognizing and targeting the persistent chronic inflammation over an extended period before attempting surgical reconstruction. This novel strategy of prolonged medical therapy to create a conducive wound environment was critical to the success of the skin graft, in which all previous interventions had failed, offering a potential new paradigm for managing this challenging sequela of snake bite."

Journal • Infectious Disease • Inflammation • Oncology • Pain

Assessing biomarkers of bone metabolism and the role of the interleukin-6 signaling pathway in patients with Duchenne muscular dystrophy. (PubMed, Neuromuscul Disord) - P2/3 | "Demographic data and total body less head (TBLH) bone mineral density (BMD) Z-scores were collected from 160 boys with DMD aged 6-11 years receiving corticosteroid therapy (prednisone, prednisolone, or deflazacort) who participated in the SPITFIRE trial (NCT03039686). Treatments to prevent osteoporotic fragility fractures in DMD are needed. Results suggest IL-6 signaling as a key mediator of bone fragility in DMD, serving as a potential new therapeutic approach."

Biomarker • Journal • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy • Musculoskeletal Diseases • Orthopedics • Osteoporosis • Rheumatology • DKK1 • IL6 • IL6R • TNFRSF11B

From case to caution: hyponatremia in a patient with Duchenne muscular dystrophy on vamorolone and lessons for clinicians. (PubMed, Neuromuscul Disord) - "We report a patient with DMD who switched from daily deflazacort to vamorolone at age 13.0 years. Six months later, he developed fever, vomiting, and diarrhea, necessitating oral hydrocortisone stress dosing...Lisinopril and spironolactone were withheld. This case raised concern for an adrenal crisis, manifested by mineralocorticoid deficiency. It highlights the importance of stress dosing and checking electrolytes during illness when on vamorolone, especially if on cardiac medications, and education of patients, families, and multidisciplinary healthcare providers."

Journal • Cardiovascular • Duchenne Muscular Dystrophy • Endocrine Disorders • Genetic Disorders • Heart Failure • Muscular Dystrophy • Nephrology • Renal Disease

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