vascular ehlers danlos syndrome with ruptured thoracic aortic aneurysm complicated by vascular graft and endograft infection and aortobronchial fistula

Original title: “Vascular Ehlers-Danlos syndrome with ruptured thoracic aortic aneurysm complicated by vascular graft and endograft infection and aortobronchial fistula.”

Three years ago, a train derailment spilled hazardous chemicals into the Ohio community.

Original title: “NIH opens East Palestine Health Research Office to study train disaster”

Real-world inotuzumab ozogamicin boosts remission in relapsed/refractory B-cell ALL, supports transplant, and flags sinusoidal obstruction syndrome risk. Reasonable remission rates were seen when inotuzumab ozogamicin was administered as a salvage therapy to patients with relapsed/refractory B-cell · acute lymphoblastic leukemia (B-ALL), with mean (SD) survival rates of 51.7% (8.1%) overall and 32.4% (8.0%) event-free, according to new research in · Blood Cancer Journal.1 These real-world data are helping to fill a data gap for such evidence currently left by limited studies in this disease and medication, which have been concentrated in clinical trial settings.2-4 Depth of response was clinically meaningful: MRD-negative status occurred in 71.4% of CR/CRi patients, enabling consolidation with ASCT or CAR T in 67.8% overall. Post-therapy hepatic SOS/veno-occlusive disease remained a major toxicity signal, with 9/10 cases after transplant and no clear correlation with cumulative dose or IO–transplant interval. European LeukemiaNet criteria. Of this group, 71.4% (n = 20) reached minimal residual disease–negative status, and 67.8% (n = 19) followed inotuzumab ozogamicin with allogeneic stem cell transplant (ASCT; n = 18) or chimeric antigen receptor (CAR) T-cell therapy (n = 1). Inotuzumab ozogamicin versus FLAG-Ida in the treatment of relapsed or refractory B-cell acute lymphoblastic leukemia – real-world resource use data. Leuk Lymphoma. 2020;61(2):491-493. doi:10.1080/10428194.2019.1672057 · Molina O, Bataller A, Thampi N, et al. Near-haploidy and low-hypodiploidy in B-cell acute lymphoblastic leukemia: when less is too much. Cancers (Basel). 2021;14(1):32. doi:10.3390/cancers14010032 · Hepatic sinusoidal obstruction syndrome (veno-occlusive disease) in adults.

Original title: “Inotuzumab Ozogamicin Outcomes in ALL Validated Outside of Clinical Trials | AJMC”

region resolved proteomic map of the human brain functional interconnections and neurological implications

Original title: “Region-resolved proteomic map of the human brain: functional interconnections and neurological implications.”

molecular insight into proliferative verrucous leukoplakia are tert promoter mutations a predictive marker

Original title: “Molecular insight into Proliferative Verrucous Leukoplakia: Are TERT promoter mutations a predictive marker?”

Prostate Cancer and Prostatic Diseases - Editorial: Advancing external beam radiotherapy—and rediscovering what brachytherapy already provides? Prostate Cancer and Prostatic Diseases (2026)Cite this article ... We read with interest the article entitled “Reducing adverse effects in hypofractionated radiotherapy for prostate cancer: Clinical evidence and emerging mitigation strategies,” and commend the authors for their comprehensive synthesis of toxicity data in this evolving field. In this systematic review, evidence from 32 phase III clinical trials was analyzed and summarized. Furthermore, other randomized trials have failed to demonstrate an excess in toxicity compared with EBRT alone [3, 4], or to report clinically meaningful levels of severe toxicity [6] comparable to those observed in ASCENDE-RT [5]. Certainly, not all patients are candidates for prostate brachytherapy or should undergo boost treatment with this modality. Finally, the utilization of brachytherapy as a boost has been demonstrated to reduce biochemical disease progression and local relapse when compared to augmented radiotherapy schedules [3,4,5], a feature just recently achieved by external beam alone, through focal boosting to the dominant lesions as nicely demonstrated in the FLAME-trial [NCT01168479]. Toxicity associated with prostate brachytherapy has been raised as a concern, leading some to forgo this treatment strategy in favor of external beam radiotherapy (EBRT) alone. In this context, we highlight the efforts of the PR24 trial for directly comparing brachytherapy boost versus SBRT in a large international phase III study [NCT06235697]. Le Guevelou J, Zilli T, Ferrario F, Murthy V, Tree A, Loblaw A, et al. Reducing adverse effects in hypofractionated radiotherapy for prostate cancer: clinical evidence and emerging mitigation strategies.

Original title: “Editorial: Advancing external beam radiotherapy—and rediscovering what brachytherapy already provides? | Prostate Cancer and Prostatic Diseases”

study of polr3a variants in a family trio suggests mutation specific pathogenetic mechanisms insights from integrative omic approaches

Original title: “Study of POLR3A variants in a family trio suggests mutation-specific pathogenetic mechanisms: insights from integrative OMIC approaches.”

effects of the phf6 gene mutation on myeloid neoplasms a single center cohort underpinned by a systematic review of literature

Original title: “The effects of the PHF6 gene mutation on myeloid neoplasms. A single-center cohort underpinned by a systematic review of literature.”

Dr. Suneel Kamath walks patients through what they need to know about their gastrointestinal cancers following major oncology meetings. So, while it certainly feels risky and scary in many ways, it is often a great way for people to get access to newer therapies before they are otherwise available. I also emphasize that being in a clinical trial is never a coercive situation. If at any point it no longer makes sense for you, you can pull out at any time. Generally, these days, we are designing these trials in a great way where there is usually some sort of biomarker or a good rationale for why a drug will work in a certain disease or for a certain person. KRAS mutations are prevalent in pancreatic cancer, and emerging targeted therapies offer promising treatment advancements. Comprehensive biomarker testing is essential, and patient advocacy can ensure appropriate testing is conducted. Clinical trials provide access to innovative therapies, with patients receiving at least the standard of care, and participation is non-coercive.SHOW MORE It’s not a rare biomarker. If we had an effective drug for this gene, that would be an effective therapy for more than 90% of people with pancreatic cancer, and that would truly be an advance. With more time, the hope is that we start to identify more targets so that we can personalize treatments for every individual patient and every individual tumor, rather than relying on untargeted regimens. The biggest thing I always try to emphasize for people regarding clinical trials in the cancer space is that you are never going to be a "guinea pig." That is often a fear many people have, and it is very understandable. In cancer clinical trials, active treatment is always given; patients receive at least the routine standard of care that we provide today.

Original title: “Personalized Care in GI Cancer and More on Biomarkers and Clinical Trials | CURE”

For people living with rare genetic bone disorders, everyday life can be unpredictable. A minor injury, a routine dental visit, even normal growth can trigger permanent bone formation that limits movement, causes chronic pain, and shortens life expectancy.A Queen’s-founded biotech startup ... For people living with rare genetic bone disorders, everyday life can be unpredictable. A minor injury, a routine dental visit, even normal growth can trigger permanent bone formation that limits movement, causes chronic pain, and shortens life expectancy.A Queen’s-founded biotech startup is now working to change that reality. A Queen’s-founded startup is translating long-term research into potential treatments for rare genetic bone disorders. By Mitchell Fox, Senior Communications Coordinator ... Martin Petkovich is the scientific founder of Ocythera and works to translate long-term academic research into new therapeutic approaches. Petkovich has founded three therapeutic companies over the course of his career, giving him experience in moving discoveries from academic research toward clinical development. Advancing the research required contributions from experts in chemistry and disease biology. Preclinical studies drew on advanced imaging and technical expertise through the Queen’s Cardio Pulmonary Unit, allowing the team to examine how abnormal bone formed and how it responded to treatment. adMare has now re-invested in the company, alongside Investissement Quebec, to support the next phases of development. Ocythera is now completing the studies required before applying to Health Canada and the U.S. Food and Drug Administration to begin human clinical trials, including safety testing, manufacturing planning, and regulatory review.

Original title: “Research driving treatment of rare diseases | Queen's Gazette”

integrative genetic and liver transcriptomic analyses identify trib1al as a target for steatotic liver disease

Original title: “Integrative genetic and liver transcriptomic analyses identify TRIB1AL as a target for steatotic liver disease.”

multiple hereditary infundibolocystic basal cell carcinoma report of a sporadic case with a novel pathogenic germline variant in sufu

Original title: “Multiple hereditary infundibolocystic basal cell carcinoma: report of a sporadic case with a novel pathogenic germline variant in SUFU.”

sarcoidosis disease mechanisms diagnostic pathway and treatment

Original title: “Sarcoidosis: Disease mechanisms, diagnostic pathway and treatment.”

sepsis and rectal injuries in patients with pelvic fractures at a level 1 trauma center a retrospective cohort study

Original title: “Sepsis and rectal injuries in patients with pelvic fractures at a level 1 trauma center: a retrospective cohort study.”

six previously undescribed bisabolane type sesquiterpenes from marine derived fungus aspergillus sp whuf04 170 and their antimicrobial activity

Original title: “Six previously undescribed bisabolane-type sesquiterpenes from marine derived fungus Aspergillus sp. WHUF04-170 and their antimicrobial activity.”

linear iga bullous dermatosis in a 4 year old child

Original title: “Linear IgA bullous dermatosis in a 4-year-old child.”

aggressive infantile melanoma arising in a congenital nevus with rare braf and bcor mutations a case report and literature review of pediatric melanoma

Original title: “Aggressive infantile melanoma arising in a congenital nevus with rare BRAF and BCOR mutations: a case report and literature review of pediatric melanoma.”

maximizing access to cell and gene therapy for patients with rare diseases

Original title: “Maximizing Access to Cell and Gene Therapy for Patients with Rare Diseases.”

infratentorial superficial siderosis a diagnostic challenge from the general practice to the hospital

Original title: “[Infratentorial superficial siderosis : a diagnostic challenge from the general practice to the hospital].”

through the lens of marketing authorization holders experience in use of real world data and real world evidence in drug development and regulatory submissions in eu

Original title: “Through the lens of marketing authorization holders: experience in use of real-world data and real-world evidence in drug development and regulatory submissions in EU.”