autoimmune osteomalacia a novel fgf23 related hypophosphatemic osteomalacia

Original title: “Autoimmune osteomalacia: a novel FGF23-related hypophosphatemic osteomalacia.”

mitochondrial energetic failure underlies flvcr1 related sensory neuropathy

Original title: “Mitochondrial energetic failure underlies FLVCR1-related sensory neuropathy.”

huntington s disease is the best investment in neuroscience today

Original title: “Huntington's disease is the best investment in neuroscience today.”

stress hyperglycemia ratio in coronary artery disease a systematic review and meta analysis of severity and prognosis

Original title: “Stress hyperglycemia ratio in coronary artery disease: a systematic review and meta-analysis of severity and prognosis.”

pyrophosphate scintigraphy changes after tafamidis therapy proposed novel index from quantitative single photon emission computed tomography

Original title: “Pyrophosphate Scintigraphy Changes After Tafamidis Therapy　- Proposed Novel Index From Quantitative Single-Photon Emission Computed Tomography.”

mucous membrane pemphigoid after anti pd 1 therapy risk stratified management and treatment outcomes

Original title: “Mucous Membrane Pemphigoid After Anti-PD-1 Therapy: Risk-Stratified Management and Treatment Outcomes.”

atp7b c 3316 g a variant is associated with mild subphenotype in wilson disease a single center cohort study

Original title: “The ATP7B c.3316 G > A variant is associated with mild subphenotype in Wilson disease: a single-center cohort study.”

clinical diagnosis and genetic analysis of a rare case of duchenne muscular dystrophy and spinal muscular atrophy

Original title: “Clinical diagnosis and genetic analysis of a rare case of Duchenne muscular dystrophy and spinal muscular atrophy.”

herpetic ocular infections in solid organ transplantation a narrative review

Original title: “Herpetic ocular infections in solid organ transplantation: A narrative review.”

shla flexneriigel 1c bacteremia in a child a case report

Original title: “Shla flexneriigel 1c bacteremia in a child: a case report.”

treatment and management of osteofibrous dysplasia like adamantinoma a single institution experience

Original title: “Treatment and Management of Osteofibrous Dysplasia Like Adamantinoma - A Single Institution Experience.”

nucleic acid drug delivery in ophthalmology emerging trends and future perspectives

Original title: “Nucleic acid drug delivery in ophthalmology: Emerging trends and future perspectives.”

erratum to deep insights and clinical benefits from the comprehensive cohort of fetal skeletal dysplasia in china

Original title: “Erratum to 'Deep insights and clinical benefits from the comprehensive cohort of fetal skeletal dysplasia in China'.”

distinct mutational signature and clonal evolution in constitutional mismatch repair deficiency associated high grade gliomas

Original title: “Distinct mutational signature and clonal evolution in constitutional mismatch repair deficiency-associated high-grade gliomas.”

splice rescue of protein truncating variants is associated with reduced selection pressure and attenuated disease phenotypes

Original title: “Splice rescue of protein-truncating variants is associated with reduced selection pressure and attenuated disease phenotypes.”

The trial lead sat in a high-stakes protocol review meeting, subtly tilting their laptop screen away from the digital transformation team. On the surface, the sponsor had just deployed a multi-million-dollar agentic AI workflow designed to automate requirement documentation and signal detection. Innovating Biothreat Therapies: An Interview with Head of GigaGen · Moe Alsumidaie · Johnson & Johnson’s Innovative Approach to Enhancing Diversity in Clinical Trials · Moe Alsumidaie · Navigating Complexities in Obesity Clinical Trials: Insights from Skye Bioscience · Moe Alsumidaie · How To Leverage eClinical Technology to Enhance Sponsor-Site Collaboration · Moe Alsumidaie · How to Navigate Rare Disease Clinical Trial Recruitment · Strategic Navigation for Clinical Trials in Mid-Size Biopharma: Insights from Almirall · Moe Alsumidaie · Navigating the Future of Endometriosis Diagnostics · Moe Alsumidaie · How Sangamo is Navigating the Future of Gene Therapy for Rare Diseases · Moe Alsumidaie · Redefining Clinical Imaging: How a Site-First Approach is Setting a New Standard for Transparency and Data Integrity ... Building Trust Through Transparency: Apellis Pharma’s Steph Viscomi on Lean TMF and Site Collaboration ... Veeva and OpenEvidence Form Strategic AI Partnership to Revolutionize Trial Access and Therapeutic Adoption Guided, Then Penalized: A Deep Dive into the FDA’s MDMA Ruling and What it Means for Psychedelic Clinical Trials ... How FDA’s Guidance on Decentralized Trials Applies to Community-based Research Execution and Compliance ... New Obesity Treatment Mechanisms: Dr.

Original title: “Why Big Pharma Clinical Trial Digital Transformation Initiatives Fail”

The prognostic implications of TP53 alterations in patients with relapsed or refractory (r/r) aggressive B-cell non-Hodgkin lymphoma (B-NHL) treated with chimeric antigen receptor (CAR) T-cell therapy remain inadequately characterized, particularly with respect to long-term outcomes. Signal Transduction and Targeted Therapy - Long-term follow-up demonstrates the curative potential of dual CD19/CD22 CAR-T-cell therapy alone or combined with autologous stem cell transplantation... Multivariate analysis identified treatment options and the presence of bulky disease as independent adverse prognostic factors for OS and PFS. These findings suggest that dual-target CD19/CD22 CAR-T-cell therapy, particularly when integrated with ASCT, may mitigate the adverse prognostic influence of TP53 alterations, offering sustained clinical benefit with manageable long-term toxicity in r/r aggressive B-NHL. This was a single-center, investigator-initiated study comprising two independent clinical trials designed to evaluate the efficacy and safety of dual-target CD19/CD22 CAR-T-cell therapy administered either as monotherapy (cohort A) or in combination with ASCT (cohort B) in patients with r/r aggressive B-NHL in whom TP53 mutation and deletions of chromosome 17p [del(17p)] were screened through next-generation sequencing or fluorescence in situ hybridization.28 The inclusion and exclusion criteria for both trials have been previously published.28 Clinical remission rates, long-term efficacy outcomes, safety parameters, and immune cell subset profiles were comprehensively evaluated in relation to disease and treatment characteristics. 5). These included hypothyroidism (n = 3) and atopic dermatitis (n = 2). Additional isolated events included immune-mediated pleural effusion, asthma, posttransplant lymphoproliferative disorder (PTLD), and coronary artery disease. All patients with these complications received appropriate therapeutic management and subsequently recovered. The incidence and distribution of these late AEs did not differ significantly between the two cohorts or different TP53 statuses (Supplementary Tables 4, 5). Univariate and multivariate Cox proportional hazards regression analyses were performed to identify clinical and treatment-related variables associated with OS and PFS in the entire study population (Table 2) or in either cohort (Supplementary Tables 6, 7).

Original title: “Long-term follow-up demonstrates the curative potential of dual CD19/CD22 CAR-T-cell therapy alone or combined with autologous stem cell transplantation in TP53-altered relapsed/refractory B-cell non-Hodgkin lymphoma | Signal Transduction and Targeted Therapy”

ultrasound in the diagnosis and management of plantar fibromatosis ledderhose disease a case report

Original title: “Ultrasound in the diagnosis and management of plantar fibromatosis (ledderhose disease): A case report.”

aortic malignancy masquerading as infrarenal aortitis a diagnostic challenge

Original title: “Aortic malignancy masquerading as infrarenal aortitis: A diagnostic challenge.”