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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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Research

Biogen's study on BIIB080 shows potential for improving cognition in Alzheimer's patients, although it raises questions about the efficacy of higher doses. This research could influence future drug development strategies in tau-targeting therapies.

The study provides the clearest evidence yet that medicines like BIIB080 might improve cognition, but leaves unclear why higher doses didn’t perform better.

BioPharma Dive·4 months ago·Read at source →

Original title: “Biogen data highlight promise, questions surrounding tau-targeting Alzheimer’s drugs”

inherited blood coagulation disorder sickle cell disease+1 moreResearch

Gene Therapy for Blood Disorders: Benefits, Risks & Future

Gene & Cell Therapy·4 months ago·Read at source →

Research

A systematic review and meta-analysis involving 15,427 patients highlights the effectiveness of JAK inhibitors in treating inflammatory skin diseases. This comprehensive study consolidates clinical trial and real-world outcomes, providing valuable insights for future research and treatment strategies.

jak inhibitors in inflammatory skin diseases a 15 427 patient systematic review and meta analysis of clinical trial and real world outcomes

PubMed Rare Disease·4 months ago·Read at source →

Original title: “JAK Inhibitors in Inflammatory Skin Diseases: A 15,427-Patient Systematic Review and Meta-analysis of Clinical Trial and Real-World Outcomes.”

angiosarcoma liver angiosarcoma+1 moreResearch

Recent research highlights improved oncologic outcomes for patients with radiation-associated angiosarcoma of the breast using trimodality therapy. This study underscores the potential benefits of combining surgery, radiation, and chemotherapy in treating this rare cancer.

improved oncologic outcomes with trimodality therapy for radiation associated angiosarcoma of the breast

PubMed Rare Disease·4 months ago·Read at source →

Original title: “Improved Oncologic Outcomes with Trimodality Therapy for Radiation-Associated Angiosarcoma of the Breast.”

Research

Shaping the future of limbal stem cell therapy: advances in substrates, techniques, and science for cultivated autologous and allogeneic limbal-epithelial transplantation.

PubMed Rare Disease·4 months ago·Read at source →

Research

Gene therapy for uveal melanoma.

PubMed Rare Disease·4 months ago·Read at source →

multiple endocrine neoplasia type 2B multiple endocrine neoplasia type 2Research

A case report highlights the coexistence of multiple endocrine neoplasia type 2B and centri-acinar emphysema in a patient with alpha-1 antitrypsin deficiency. This finding may provide insights into the complex interactions between these rare conditions.

coexistence of multiple endocrine neoplasia type 2b and centri acinar emphysema with alpha 1 antitrypsin deficiency a case report

PubMed Rare Disease·4 months ago·Read at source →

Original title: “Coexistence of Multiple Endocrine Neoplasia Type 2B and Centri-Acinar Emphysema With Alpha-1 Antitrypsin Deficiency: A Case Report.”

Research

A recent publication discusses the current applications and challenges of artificial intelligence in clinical genetics. The review highlights how AI can enhance genetic analysis but also addresses the ethical and technical hurdles that remain.

artificial intelligence in clinical genetics current applications and challenges

PubMed Rare Disease·4 months ago·Read at source →

Original title: “Artificial Intelligence in Clinical Genetics: Current Applications and Challenges.”

Research

A recent cohort study investigates the impact of travel distance on hospital utilization among children requiring respiratory support. The findings highlight potential barriers to care that could inform healthcare access strategies.

too far to care a cohort study on travel distance and hospital use in children living with respiratory support

PubMed Rare Disease·4 months ago·Read at source →

Original title: “Too far to care? A cohort study on travel distance and hospital use in children living with respiratory support.”

Research

Recent research published in PubMed reveals the structural polymorphism of polyG inclusions through in situ cryo-electron tomography. This study enhances the understanding of polyG structures, which may have implications for related rare diseases.

structural polymorphism of polyg inclusions revealed by in situ cryo electron tomography

PubMed Rare Disease·4 months ago·Read at source →

Original title: “Structural Polymorphism of polyG Inclusions Revealed by In Situ Cryo-Electron Tomography.”

salivary gland acinic cell carcinoma parotid gland acinic cell carcinoma+1 moreResearch

A recent analysis of the National Cancer Database highlights the clinicopathologic features and treatment patterns of acinic cell carcinoma of the breast. The study provides insights into survival outcomes, contributing valuable data to the understanding of this rare breast cancer subtype.

clinicopathologic features treatment patterns and survival outcomes of acinic cell carcinoma of the breast a national cancer database analysis

PubMed Rare Disease·4 months ago·Read at source →

Original title: “Clinicopathologic features, treatment patterns and survival outcomes of acinic cell carcinoma of the breast: a national cancer database analysis.”

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

A recent study explores the implications of anifrolumab treatment during pregnancy and lactation in patients with systemic lupus erythematosus. The findings contribute to understanding the safety profile of this therapy in reproductive health contexts.

pregnancy and lactation during anifrolumab treatment in systemic lupus erythematosus

PubMed Rare Disease·4 months ago·Read at source →

Original title: “Pregnancy and lactation during anifrolumab treatment in systemic lupus erythematosus.”

lipoma psoriatic arthritisResearch

A case study highlights bilateral knee lipoma arborescens that mimicked persistent inflammatory arthritis in a patient with psoriatic arthritis. This discovery may enhance understanding of differential diagnoses in inflammatory conditions.

bilateral knee lipoma arborescens mimicking persistent inflammatory arthritis in a patient with psoriatic arthritis

PubMed Rare Disease·4 months ago·Read at source →

Original title: “Bilateral knee lipoma arborescens mimicking persistent inflammatory arthritis in a patient with psoriatic arthritis.”

Research

A new study discusses nonparametric testing methods for evaluating relative effects in non-inferiority clinical trials involving multiple experimental drugs. This research could enhance statistical approaches in rare disease drug development.

nonparametric testing methods based on relative effect in non inferiority clinical trial with multiple experimental drugs

PubMed Rare Disease·4 months ago·Read at source →

Original title: “Nonparametric testing methods based on relative effect in non-inferiority clinical trial with multiple experimental drugs.”

adrenocortical carcinoma, hereditary pheochromocytoma+1 moreResearch

Recent research highlights systemic management strategies for adrenal malignancies, specifically adrenocortical carcinoma and pheochromocytoma/paraganglioma. This study contributes to the understanding of treatment approaches for these rare cancers.

systemic management of adrenal malignancies adrenocortical carcinoma and pheochromocytoma paraganglioma

PubMed Rare Disease·4 months ago·Read at source →

Original title: “Systemic Management of Adrenal Malignancies: Adrenocortical carcinoma and pheochromocytoma/paraganglioma.”

Research

A recent study investigates the CD38-associated surfaceome using nanobody-targeted TurboID technology. This research could enhance understanding of CD38's role in various diseases, potentially leading to new therapeutic strategies.

exploring the cd38 associated surfaceome via nanobody targeted turboid

PubMed Rare Disease·4 months ago·Read at source →

Original title: “Exploring the CD38-associated surfaceome via nanobody-targeted TurboID.”

inflammatory bowel disease inflammatory bowel disease 10+1 moreResearch

A case report highlights gastrointestinal basidiobolomycosis presenting symptoms similar to inflammatory bowel disease, providing valuable insights into its diagnosis and management. This review of Saudi literature emphasizes the need for awareness of this rare fungal infection.

case report on gastrointestinal basidiobolomycosis mimicking inflammatory bowel disease insights and review of saudi literature

PubMed Rare Disease·4 months ago·Read at source →

Original title: “Case Report on Gastrointestinal Basidiobolomycosis Mimicking Inflammatory Bowel Disease: Insights and Review of Saudi Literature.”

Research

A multicenter cohort study evaluates the effectiveness of traditional versus novel immune-inflammatory indices in measuring disease activity in Sjögren's disease. The findings could influence future assessment strategies for this condition.

comparative value of traditional versus novel immune inflammatory indices in assessing disease activity of sj gren s disease a multicenter cohort study

PubMed Rare Disease·4 months ago·Read at source →

Original title: “Comparative Value of Traditional versus Novel Immune-Inflammatory Indices in Assessing Disease Activity of Sjögren's Disease: A Multicenter Cohort Study.”

Research

A systematic scoping review highlights the evaluation of diagnostic and therapeutic technologies for ultra-rare diseases. This evidence mapping identifies key concepts, methods, and outcomes, providing a foundation for future research and development.

evidence mapping of concepts methods and outcomes in the evaluation of diagnostic and therapeutic technologies for ultra rare diseases a systematic scoping review

PubMed Rare Disease·4 months ago·Read at source →

Original title: “Evidence mapping of concepts, methods, and outcomes in the evaluation of diagnostic and therapeutic technologies for ultra-rare diseases: a systematic scoping review.”

acute posterior multifocal placoid pigment epitheliopathyResearch

A study highlights the occurrence of macular neovascular membranes as a late complication of acute posterior multifocal placoid pigment epitheliopathy (APMPPE), treated effectively with intravitreal aflibercept. This research contributes to understanding the long-term management of APMPPE.

macular neovascular membrane as a late complication of acute posterior multifocal placoid pigment epitheliopathy apmppe managed with intravitreal aflibercept

PubMed Rare Disease·4 months ago·Read at source →

Original title: “Macular Neovascular Membrane as a Late Complication of Acute Posterior Multifocal Placoid Pigment Epitheliopathy (APMPPE) Managed With Intravitreal Aflibercept.”

Previous235 / 485Next

Daily Briefings

Not rare diseases news,news about your rare disease.

Earlier

Biogen's study on BIIB080 shows potential for improving cognition in Alzheimer's patients, although it raises questions about the efficacy of higher doses. This research could influence future drug development strategies in tau-targeting therapies.

Gene Therapy for Blood Disorders: Benefits, Risks & Future

Recent research highlights improved oncologic outcomes for patients with radiation-associated angiosarcoma of the breast using trimodality therapy. This study underscores the potential benefits of combining surgery, radiation, and chemotherapy in treating this rare cancer.

Shaping the future of limbal stem cell therapy: advances in substrates, techniques, and science for cultivated autologous and allogeneic limbal-epithelial transplantation.

Gene therapy for uveal melanoma.

A case report highlights the coexistence of multiple endocrine neoplasia type 2B and centri-acinar emphysema in a patient with alpha-1 antitrypsin deficiency. This finding may provide insights into the complex interactions between these rare conditions.

A recent publication discusses the current applications and challenges of artificial intelligence in clinical genetics. The review highlights how AI can enhance genetic analysis but also addresses the ethical and technical hurdles that remain.

A recent cohort study investigates the impact of travel distance on hospital utilization among children requiring respiratory support. The findings highlight potential barriers to care that could inform healthcare access strategies.

Recent research published in PubMed reveals the structural polymorphism of polyG inclusions through in situ cryo-electron tomography. This study enhances the understanding of polyG structures, which may have implications for related rare diseases.

A recent analysis of the National Cancer Database highlights the clinicopathologic features and treatment patterns of acinic cell carcinoma of the breast. The study provides insights into survival outcomes, contributing valuable data to the understanding of this rare breast cancer subtype.

A recent study explores the implications of anifrolumab treatment during pregnancy and lactation in patients with systemic lupus erythematosus. The findings contribute to understanding the safety profile of this therapy in reproductive health contexts.

A case study highlights bilateral knee lipoma arborescens that mimicked persistent inflammatory arthritis in a patient with psoriatic arthritis. This discovery may enhance understanding of differential diagnoses in inflammatory conditions.

A new study discusses nonparametric testing methods for evaluating relative effects in non-inferiority clinical trials involving multiple experimental drugs. This research could enhance statistical approaches in rare disease drug development.

Recent research highlights systemic management strategies for adrenal malignancies, specifically adrenocortical carcinoma and pheochromocytoma/paraganglioma. This study contributes to the understanding of treatment approaches for these rare cancers.

A recent study investigates the CD38-associated surfaceome using nanobody-targeted TurboID technology. This research could enhance understanding of CD38's role in various diseases, potentially leading to new therapeutic strategies.

A case report highlights gastrointestinal basidiobolomycosis presenting symptoms similar to inflammatory bowel disease, providing valuable insights into its diagnosis and management. This review of Saudi literature emphasizes the need for awareness of this rare fungal infection.

A multicenter cohort study evaluates the effectiveness of traditional versus novel immune-inflammatory indices in measuring disease activity in Sjögren's disease. The findings could influence future assessment strategies for this condition.

A systematic scoping review highlights the evaluation of diagnostic and therapeutic technologies for ultra-rare diseases. This evidence mapping identifies key concepts, methods, and outcomes, providing a foundation for future research and development.

A study highlights the occurrence of macular neovascular membranes as a late complication of acute posterior multifocal placoid pigment epitheliopathy (APMPPE), treated effectively with intravitreal aflibercept. This research contributes to understanding the long-term management of APMPPE.

Not rare diseases news,
news about your rare disease.