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What's new in your rare disease | Kisho

Not rare diseases news,
news about your rare disease.

Every story we pull is tagged to the disease it’s about — so you can filter to your condition and read only what matters.

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differentiated thyroid carcinoma poorly differentiated thyroid gland carcinoma+1 moreResearch

A new graded prognostic assessment for brain metastases in differentiated thyroid carcinoma has been developed and validated using data from over 10,000 patients. This tool aims to improve patient stratification and treatment planning.

differentiated thyroid carcinoma specific graded prognostic assessment for brain metastases derivation in a 10 306 patient cohort and external validation

PubMed Rare Disease·7 months ago·Read at source →

Original title: “A differentiated thyroid carcinoma-specific graded prognostic assessment for brain metastases: derivation in a 10,306-patient cohort and external validation.”

post-transplant lymphoproliferative diseaseResearch

A recent case study highlights an uncommon presentation of post-transplant lymphoproliferative disorder in a child following liver transplantation, characterized by massive splenomegaly. This finding contributes to the understanding of rare disease manifestations in pediatric transplant patients.

uncommon presentation of a rare disease post transplant lymphoproliferative disorder in a child post liver transplant presenting as massive splenomegaly

PubMed Rare Disease·7 months ago·Read at source →

Original title: “Uncommon Presentation of a Rare Disease: Post-Transplant Lymphoproliferative Disorder in a Child Post Liver Transplant Presenting as Massive Splenomegaly.”

Research

The European Reference Networks (ERNs) initiative aims to enhance collaboration and knowledge sharing in the field of rare and complex diseases from 2017 to 2025. This program is designed to improve patient care and access to specialized treatments across Europe.

european reference networks a flagship activity of the eu in the field of rare and complex diseases from 2017 to 2025

PubMed Rare Disease·7 months ago·Read at source →

Original title: “European Reference Networks - a flagship activity of the EU in the field of rare and complex diseases: from 2017 to 2025.”

Research

A new study explores the use of deep contrastive learning for predicting phenotypes associated with missense variants. This research could enhance understanding of genetic variations and their implications in rare diseases.

phenotypic prediction of missense variants via deep contrastive learning

PubMed Rare Disease·7 months ago·Read at source →

Original title: “Phenotypic prediction of missense variants via deep contrastive learning.”

myasthenia gravis transient neonatal myasthenia gravisResearch

A recent study highlights the inadequacy of current immunosuppressive treatments for myasthenia gravis, suggesting a need for new therapeutic approaches. The findings underscore the potential for innovative treatments to improve patient outcomes.

insufficient immunosuppressive treatment in patients with myasthenia gravis in the context of the possibility of new therapies a single center experience

PubMed Rare Disease·7 months ago·Read at source →

Original title: “Insufficient immunosuppressive treatment in patients with myasthenia gravis in the context of the possibility of new therapies - a single center experience.”

nasopharyngeal carcinomaResearch

Recent research highlights the distinct features and favorable prognosis of childhood nasopharyngeal carcinoma, a rare disease. This study contributes to the understanding of its clinical characteristics and potential treatment pathways.

childhood nasopharyngeal carcinoma a rare disease with distinct features and favorable prognosis

PubMed Rare Disease·7 months ago·Read at source →

Original title: “Childhood Nasopharyngeal Carcinoma: A Rare Disease with Distinct Features and Favorable Prognosis.”

bacteremia, susceptibilityResearch

A case study highlights the potential risks associated with the probiotic Alkalihalobacillus clausii, demonstrating its link to bacteremia in a child. This finding raises important questions about the safety of probiotics in vulnerable populations.

double edged sword of probiotic alkalihalobacillus clausii bacteremia in a child

PubMed Rare Disease·7 months ago·Read at source →

Original title: “A Double-Edged Sword of Probiotic: Alkalihalobacillus clausii Bacteremia in a Child.”

Research

A study published on PubMed presents a comparative proteomic profiling of clinical Klebsiella pneumoniae strains and their outer membrane vesicles. This research may provide insights into the pathogenic mechanisms of Klebsiella pneumoniae, a significant cause of infections.

comparative proteomic profiling of clinical klebsiella pneumoniae strains and the corresponding outer membrane vesicles

PubMed Rare Disease·7 months ago·Read at source →

Original title: “Comparative Proteomic Profiling of Clinical Klebsiella pneumoniae Strains and the Corresponding Outer Membrane Vesicles.”

nut midline carcinomaResearch

A case report details a downhill esophageal variceal rupture linked to superior vena cava syndrome in a patient with NUT carcinoma. This finding contributes to the understanding of complications associated with this rare cancer.

downhill esophageal variceal rupture caused by superior vena cava syndrome in nut carcinoma a case report

PubMed Rare Disease·7 months ago·Read at source →

Original title: “Downhill Esophageal Variceal Rupture Caused by Superior Vena Cava Syndrome in NUT Carcinoma: A Case Report.”

hyperinsulinismResearch

A retrospective review highlights the natural history of hyperinsulinism hyperammonemia syndrome, incorporating patient-centered data. This study provides valuable insights into the disease's progression and patient experiences.

natural history of the hyperinsulinism hyperammonemia syndrome a retrospective review incorporating patient centered data

PubMed Rare Disease·7 months ago·Read at source →

Original title: “Natural History of the Hyperinsulinism Hyperammonemia Syndrome - A Retrospective Review Incorporating Patient-centered Data.”

von Willebrand disease 3 platelet-type von Willebrand disease+1 moreResearch

Recent publication details two clinical cases of Type 3 Von Willebrand disease, a rare bleeding disorder. The study contributes to the understanding of this condition and its clinical manifestations.

type 3 von willebrand disease two clinical cases of a rare disorder

PubMed Rare Disease·7 months ago·Read at source →

Original title: “Type 3 Von Willebrand disease: two clinical cases of a rare disorder.”

systemic lupus erythematosus systemic lupus erythematosus related to C1QA+1 moreResearch

A real-world study comparing tofacitinib and BAFF inhibitors in patients with mild to moderate systemic lupus erythematosus provides new insights into treatment efficacy. The findings are based on data from the CSTAR cohort, contributing to the understanding of therapeutic options for this condition.

tofacitinib versus baff inhibitor in mild to moderate systemic lupus erythematosus a real world study based on cstar cohort

PubMed Rare Disease·7 months ago·Read at source →

Original title: “Tofacitinib versus BAFF inhibitor in mild to moderate systemic lupus erythematosus: a real-world study based on CSTAR cohort.”

squamous cell carcinoma squamous cell carcinoma of penis+1 moreResearch

A recent study explores the cellular origins and etiological factors contributing to squamous cell carcinoma of the bladder. This research provides insights that could inform future therapeutic strategies for this cancer type.

cellular origins and etiological factors for squamous cell carcinoma and related cancer types of the bladder

PubMed Rare Disease·7 months ago·Read at source →

Original title: “Cellular origins and etiological factors for squamous cell carcinoma and related cancer types of the bladder.”

Joubert syndrome Joubert syndrome 23Research

A case study highlights a 7-year-old girl with Joubert Syndrome and an NPC1 mutation, presenting with neuromotor developmental delay and ataxia. This case contributes to the understanding of the clinical manifestations associated with these rare genetic conditions.

case of joubert syndrome and npc1 mutation in a 7 year old girl presented with neuromotor developmental delay and ataxia

PubMed Rare Disease·7 months ago·Read at source →

Original title: “A case of Joubert Syndrome and NPC1 mutation in a 7-year-old girl: presented with neuromotor developmental delay and ataxia.”

Research

A systematic review examines IgM nephropathy, questioning whether it is a distinct nephropathy or part of the podocytopathy spectrum. This analysis contributes to the ongoing debate in nephrology regarding the classification and understanding of this condition.

igm nephropathy is a controversial entity a distinct nephropathy or part of the podocytopathy spectrum a systematic review of the literature

PubMed Rare Disease·7 months ago·Read at source →

Original title: “IgM nephropathy is a controversial entity: a distinct nephropathy or part of the podocytopathy spectrum? A systematic review of the literature.”

Research

A recent case report highlights the association between oral AA amyloidosis and granulomatous cheilitis, contributing to the understanding of these rare conditions. The literature review provides insights into clinical presentations and management strategies.

oral aa amyloidosis with granulomatous cheilitis a case report and literature review

PubMed Rare Disease·7 months ago·Read at source →

Original title: “Oral AA amyloidosis with granulomatous cheilitis: a case report and literature review.”

retinitis pigmentosa retinitis pigmentosa-intellectual disability-deafness-hypogenitalism syndromeResearch

A case report highlights persistent bilateral optic disc swelling in a patient with non-syndromic retinitis pigmentosa, contributing to the understanding of ocular manifestations in this condition. This finding may inform future research and clinical approaches to retinitis pigmentosa.

persistent bilateral optic disc swelling in non syndromic retinitis pigmentosa a case report

PubMed Rare Disease·7 months ago·Read at source →

Original title: “Persistent Bilateral Optic Disc Swelling in Non-Syndromic Retinitis Pigmentosa: A Case Report.”

acute myeloid leukemiaResearch

A first-in-human case report explores the repurposing of all-trans retinoic acid (ATRA) for treating AML1-MTG16⁺ acute myeloid leukemia. This study provides a translational roadmap aimed at addressing the challenges associated with rare disease treatments.

mechanism driven repurposing of all trans retinoic acid atra for aml1 mtg16 acute myeloid leukemia a first in human case report and translational roadmap to overcome the long tail barrier

PubMed Rare Disease·7 months ago·Read at source →

Original title: “Mechanism-Driven Repurposing of All-Trans Retinoic Acid (ATRA) for AML1-MTG16⁺ Acute Myeloid Leukemia: A First-in-Human Case Report and Translational Roadmap to Overcome the "Long-Tail" Barrier.”

Research

A recent study published in PubMed explores the mutational burden in patients with os odontoideum, providing insights into the genetic factors associated with this rare condition. The findings may inform future research and therapeutic approaches.

mutational burden in os odontoideum patients

PubMed Rare Disease·7 months ago·Read at source →

Original title: “The mutational burden in os odontoideum patients.”

lymphoma orbit lymphomaResearch

Initial trial results from Allogene suggest that their 'off-the-shelf' CAR-T therapy may effectively delay relapse in lymphoma patients. This breakthrough could position the therapy as a significant option in lymphoma care.

Initial results from a high-stakes trial indicate the biotech may have finally found a niche in lymphoma care for a donor-derived cell therapy.

BioPharma Dive·7 months ago·Read at source →

Original title: “Allogene data suggest ‘off-the-shelf’ CAR-T could delay relapse in lymphoma”

Previous368 / 488Next

Daily Briefings

Not rare diseases news,news about your rare disease.

Earlier

A new graded prognostic assessment for brain metastases in differentiated thyroid carcinoma has been developed and validated using data from over 10,000 patients. This tool aims to improve patient stratification and treatment planning.

A recent case study highlights an uncommon presentation of post-transplant lymphoproliferative disorder in a child following liver transplantation, characterized by massive splenomegaly. This finding contributes to the understanding of rare disease manifestations in pediatric transplant patients.

The European Reference Networks (ERNs) initiative aims to enhance collaboration and knowledge sharing in the field of rare and complex diseases from 2017 to 2025. This program is designed to improve patient care and access to specialized treatments across Europe.

A new study explores the use of deep contrastive learning for predicting phenotypes associated with missense variants. This research could enhance understanding of genetic variations and their implications in rare diseases.

A recent study highlights the inadequacy of current immunosuppressive treatments for myasthenia gravis, suggesting a need for new therapeutic approaches. The findings underscore the potential for innovative treatments to improve patient outcomes.

Recent research highlights the distinct features and favorable prognosis of childhood nasopharyngeal carcinoma, a rare disease. This study contributes to the understanding of its clinical characteristics and potential treatment pathways.

A case study highlights the potential risks associated with the probiotic Alkalihalobacillus clausii, demonstrating its link to bacteremia in a child. This finding raises important questions about the safety of probiotics in vulnerable populations.

A study published on PubMed presents a comparative proteomic profiling of clinical Klebsiella pneumoniae strains and their outer membrane vesicles. This research may provide insights into the pathogenic mechanisms of Klebsiella pneumoniae, a significant cause of infections.

A case report details a downhill esophageal variceal rupture linked to superior vena cava syndrome in a patient with NUT carcinoma. This finding contributes to the understanding of complications associated with this rare cancer.

A retrospective review highlights the natural history of hyperinsulinism hyperammonemia syndrome, incorporating patient-centered data. This study provides valuable insights into the disease's progression and patient experiences.

Recent publication details two clinical cases of Type 3 Von Willebrand disease, a rare bleeding disorder. The study contributes to the understanding of this condition and its clinical manifestations.

A recent study explores the cellular origins and etiological factors contributing to squamous cell carcinoma of the bladder. This research provides insights that could inform future therapeutic strategies for this cancer type.

A case study highlights a 7-year-old girl with Joubert Syndrome and an NPC1 mutation, presenting with neuromotor developmental delay and ataxia. This case contributes to the understanding of the clinical manifestations associated with these rare genetic conditions.

A systematic review examines IgM nephropathy, questioning whether it is a distinct nephropathy or part of the podocytopathy spectrum. This analysis contributes to the ongoing debate in nephrology regarding the classification and understanding of this condition.

A recent case report highlights the association between oral AA amyloidosis and granulomatous cheilitis, contributing to the understanding of these rare conditions. The literature review provides insights into clinical presentations and management strategies.

A case report highlights persistent bilateral optic disc swelling in a patient with non-syndromic retinitis pigmentosa, contributing to the understanding of ocular manifestations in this condition. This finding may inform future research and clinical approaches to retinitis pigmentosa.

A first-in-human case report explores the repurposing of all-trans retinoic acid (ATRA) for treating AML1-MTG16⁺ acute myeloid leukemia. This study provides a translational roadmap aimed at addressing the challenges associated with rare disease treatments.

A recent study published in PubMed explores the mutational burden in patients with os odontoideum, providing insights into the genetic factors associated with this rare condition. The findings may inform future research and therapeutic approaches.

Initial trial results from Allogene suggest that their 'off-the-shelf' CAR-T therapy may effectively delay relapse in lymphoma patients. This breakthrough could position the therapy as a significant option in lymphoma care.

Not rare diseases news,
news about your rare disease.