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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

FDA reconsiders rejected rare-disease gene therapy

Gene & Cell Therapy·5 months ago·Read at source →

rareResearch

In another reversal for rare disease field, FDA to reconsider Regenxbio gene therapy | Fierce Biotech

Gene & Cell Therapy·5 months ago·Read at source →

Cockayne syndrome disease of the tendonResearch

First Patient in the World Treated With Gene Therapy for Cockayne Syndrome

Community & Advocacy·5 months ago·Read at source →

chronic renal failure syndrome action myoclonus-renal failure syndrome+1 moreResearch

A recent study explores factors affecting the acceptance of deceased donor kidney transplant offers in children. The findings may inform strategies to improve transplant outcomes and decision-making processes.

factors influencing the acceptance of deceased donor kidney transplant offers in children a vignette survey

PubMed Rare Disease·5 months ago·Read at source →

Original title: “Factors influencing the acceptance of deceased donor kidney transplant offers in children-a vignette survey.”

peripheral arterial occlusive disease 1 peripheral vascular diseaseResearch

A recent study highlights cases of fulminant liver dysfunction following endovascular pharmacomechanical thrombectomy in patients with peripheral arterial occlusive disease. This research underscores the need for careful monitoring of liver function in these patients post-procedure.

fulminant liver dysfunction after endovascular pharmacomechanical thrombectomy for peripheral arterial occlusive disease

PubMed Rare Disease·5 months ago·Read at source →

Original title: “Fulminant liver dysfunction after endovascular pharmacomechanical thrombectomy for peripheral arterial occlusive disease.”

pyoderma gangrenosum pyoderma gangrenosum-acne-suppurative hidradenitis syndromeResearch

A recent study highlights that lesional pruritus is present in patients with pyoderma gangrenosum, although it is not the predominant symptom. This finding may influence clinical assessments and management strategies for this rare skin condition.

lesional pruritus in pyoderma gangrenosum present but not predominant

PubMed Rare Disease·5 months ago·Read at source →

Original title: “Lesional pruritus in pyoderma gangrenosum: Present but not predominant.”

Research

How Rare Disease Foundations Drive Medical Advancements

Community & Advocacy·5 months ago·Read at source →

Research

Gene Therapy Shows Promise for Life-Threatening Inherited Heart Disease | Newsroom | Weill Cornell Medicine

Gene & Cell Therapy·5 months ago·Read at source →

Research

A new publication discusses advancements in understanding rare tumors, focusing on their unique biological characteristics and potential therapeutic targets. This research highlights the need for tailored treatment approaches in managing these complex conditions.

rare tumors part two

PubMed Rare Disease·5 months ago·Read at source →

Original title: “Rare Tumors: Part Two.”

Research

A new publication discusses the complexities and challenges associated with rare tumors, highlighting the need for further research in this area. The study emphasizes the importance of understanding the unique biological characteristics of these tumors to improve diagnosis and treatment.

rare tumors part one

PubMed Rare Disease·5 months ago·Read at source →

Original title: “Rare Tumors: Part One.”

Langerhans cell histiocytosis Langerhans cell histiocytosis specific to adulthood+1 moreResearch

A case series published in PubMed highlights two patients with Nail Langerhans Cell Histiocytosis, showcasing both single-system and multisystem disease presentations. The study emphasizes the unique nail lesions associated with this rare condition.

nail langerhans cell histiocytosis a rare case series of two patients with single system and multisystem disease presenting with nail lesions

PubMed Rare Disease·5 months ago·Read at source →

Original title: “Nail Langerhans Cell Histiocytosis: A Rare Case Series of Two Patients With Single-System and Multisystem Disease Presenting With Nail Lesions.”

sickle cell disease sickle cell-hemoglobin d disease syndrome+1 moreResearch

Gene Therapy for Sickle Cell Disease: McKenzie Imagines a Future Without Pain Crises | Children's Hospital Los Angeles

Gene & Cell Therapy·5 months ago·Read at source →

Research

Intellia’s gene editor ‘keeps pace’ with Ionis in hereditary angioedema

BioSpace·5 months ago·Read at source →

malaria Plasmodium vivax malaria+1 moreResearch

A case report details a successful vaginal delivery in a patient with very severe malarial thrombocytopenia, highlighting the complexities of managing pregnancy in the context of severe malaria. This case contributes to the understanding of maternal health challenges associated with malaria.

successful vaginal delivery in very severe malarial thrombocytopenia a case report

PubMed Rare Disease·5 months ago·Read at source →

Original title: “Successful vaginal delivery in very severe malarial thrombocytopenia: a case report.”

activated PI3K-delta syndromeResearch

A new review highlights the potential of PI3K/AKT/mTOR inhibitors derived from traditional Chinese medicine for treating activated phosphoinositide 3-kinase δ syndrome. This research could pave the way for novel therapeutic strategies in managing this rare disease.

review of pi3k akt mtor inhibitors from traditional chinese medicine potential and perspective for activated phosphoinositide 3 kinase syndrome

PubMed Rare Disease·5 months ago·Read at source →

Original title: “A review of PI3K/AKT/mTOR inhibitors from traditional Chinese medicine: potential and perspective for activated phosphoinositide 3-kinase δ syndrome.”

chronic recurrent multifocal osteomyelitis inflammatory bowel disease+1 moreResearch

A French national cohort study investigates the relationship between chronic nonbacterial osteomyelitis and inflammatory bowel disease in children. This research adds to the understanding of these conditions and their potential overlap in pediatric patients.

chronic nonbacterial osteomyelitis and inflammatory bowel disease in children a french national cohort

PubMed Rare Disease·5 months ago·Read at source →

Original title: “Chronic nonbacterial osteomyelitis and inflammatory bowel disease in children: A French national cohort.”

Research

A recent study published in PubMed highlights the results of copy number variations (CNVs) analysis in patients with neurodevelopmental delay, dysmorphic features, and congenital defects. This research contributes to understanding the genetic underpinnings of these conditions.

results of cnvs analysis in patients with neurodevelopmental delay dysmorphic features and congenital defects

PubMed Rare Disease·5 months ago·Read at source →

Original title: “The results of CNVs analysis in patients with neurodevelopmental delay, dysmorphic features and congenital defects.”

breast cancer colorectal carcinomaResearch

Recent research highlights the phenomenon of breast cancer metastasizing to the colon, which can mimic primary colorectal carcinoma. This discovery may impact diagnostic approaches and treatment strategies for patients with metastatic breast cancer.

breast cancer metastasis to the colon mimicking primary colorectal carcinoma

PubMed Rare Disease·5 months ago·Read at source →

Original title: “Breast cancer metastasis to the colon mimicking primary colorectal carcinoma.”

Robinow syndrome robinow syndrome, autosomal recessive 2+1 moreResearch

A novel pathogenic variant in the DVL1 gene has been identified and functionally characterized in patients with Robinow syndrome. This discovery enhances the understanding of the genetic basis of this rare condition.

identification and functional characterization of a novel pathogenic dvl1 gene variant in robinow syndrome

PubMed Rare Disease·5 months ago·Read at source →

Original title: “Identification and functional characterization of a novel pathogenic DVL1 gene variant in Robinow syndrome.”

Netherton syndromeResearch

Recent research highlights that in-depth curation of the Human Phenotype Ontology significantly enhances prioritization performance for Netherton Syndrome. This advancement could improve diagnostic accuracy and patient outcomes for those affected by this rare condition.

in depth human phenotype ontology curation boosts prioritization performance for netherton syndrome

PubMed Rare Disease·5 months ago·Read at source →

Original title: “In-depth Human Phenotype Ontology Curation Boosts Prioritization Performance for Netherton Syndrome.”

Previous243 / 486Next

Daily Briefings

Not rare diseases news,news about your rare disease.

Earlier

FDA reconsiders rejected rare-disease gene therapy

In another reversal for rare disease field, FDA to reconsider Regenxbio gene therapy | Fierce Biotech

First Patient in the World Treated With Gene Therapy for Cockayne Syndrome

A recent study explores factors affecting the acceptance of deceased donor kidney transplant offers in children. The findings may inform strategies to improve transplant outcomes and decision-making processes.

A recent study highlights cases of fulminant liver dysfunction following endovascular pharmacomechanical thrombectomy in patients with peripheral arterial occlusive disease. This research underscores the need for careful monitoring of liver function in these patients post-procedure.

A recent study highlights that lesional pruritus is present in patients with pyoderma gangrenosum, although it is not the predominant symptom. This finding may influence clinical assessments and management strategies for this rare skin condition.

How Rare Disease Foundations Drive Medical Advancements

Gene Therapy Shows Promise for Life-Threatening Inherited Heart Disease | Newsroom | Weill Cornell Medicine

A new publication discusses advancements in understanding rare tumors, focusing on their unique biological characteristics and potential therapeutic targets. This research highlights the need for tailored treatment approaches in managing these complex conditions.

A new publication discusses the complexities and challenges associated with rare tumors, highlighting the need for further research in this area. The study emphasizes the importance of understanding the unique biological characteristics of these tumors to improve diagnosis and treatment.

A case series published in PubMed highlights two patients with Nail Langerhans Cell Histiocytosis, showcasing both single-system and multisystem disease presentations. The study emphasizes the unique nail lesions associated with this rare condition.

Gene Therapy for Sickle Cell Disease: McKenzie Imagines a Future Without Pain Crises | Children's Hospital Los Angeles

Intellia’s gene editor ‘keeps pace’ with Ionis in hereditary angioedema

A case report details a successful vaginal delivery in a patient with very severe malarial thrombocytopenia, highlighting the complexities of managing pregnancy in the context of severe malaria. This case contributes to the understanding of maternal health challenges associated with malaria.

A new review highlights the potential of PI3K/AKT/mTOR inhibitors derived from traditional Chinese medicine for treating activated phosphoinositide 3-kinase δ syndrome. This research could pave the way for novel therapeutic strategies in managing this rare disease.

A French national cohort study investigates the relationship between chronic nonbacterial osteomyelitis and inflammatory bowel disease in children. This research adds to the understanding of these conditions and their potential overlap in pediatric patients.

A recent study published in PubMed highlights the results of copy number variations (CNVs) analysis in patients with neurodevelopmental delay, dysmorphic features, and congenital defects. This research contributes to understanding the genetic underpinnings of these conditions.

Recent research highlights the phenomenon of breast cancer metastasizing to the colon, which can mimic primary colorectal carcinoma. This discovery may impact diagnostic approaches and treatment strategies for patients with metastatic breast cancer.

A novel pathogenic variant in the DVL1 gene has been identified and functionally characterized in patients with Robinow syndrome. This discovery enhances the understanding of the genetic basis of this rare condition.

Recent research highlights that in-depth curation of the Human Phenotype Ontology significantly enhances prioritization performance for Netherton Syndrome. This advancement could improve diagnostic accuracy and patient outcomes for those affected by this rare condition.

Not rare diseases news,
news about your rare disease.