Explore Diseases Find Orgs Patient Assistance News Insights About

Kisho

Kisho is an information platform, not a medical provider. Nothing on this site constitutes medical advice, diagnosis, or treatment recommendations. All content is aggregated from publicly available sources (including ClinicalTrials.gov, PubMed, FDA.gov, and Orphanet) and is provided for informational purposes only. Clinical trial eligibility, treatment decisions, and any health-related actions should always be discussed with a qualified healthcare professional. Kisho does not endorse any specific therapy, organization, or clinical trial. Terms of use · Privacy policy

Open rare disease resources — free, sourced, never gated.

Resources

Rare Disease Directory
Patient Organization Directory
Patient Assistance Programs
Rare Disease News
Clinical Trials
Research Publications

Legal

Privacy Policy
Terms of Service
Accessibility
About & Methodology
Contact

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.

News sourcesFDA·NIH·CDC·EMA·PubMed·Federal Register·NORD·EURORDIS·Global Genes·The Mighty·BioPharma Dive·BioSpace

Newsroom9,738 Trends

Daily Briefings

20 earlier

Filter & sort

Source

Sort by

Earlier

20 stories

Hurler syndrome lysosomal storage disease+1 moreResearch

Analysis of Brain Tumor Following AAV Gene Therapy Identifies Vector Integration and Reinforces Importance of Long-Term Monitoring | Children's Hospital of Philadelphia

Treatment & Clinical News·6 months ago·Read at source →

obesity disorderResearch

Recent trial results indicate that transitioning from high-dose GLP-1 injectables to lower doses or oral formulations may enhance long-term weight maintenance in individuals with obesity. These findings could influence future treatment strategies in obesity management.

A pair of trial results published in medical journals suggest switching from high-dose injectables to lower doses or pills could help people with obesity keep their weight down over the long term.

BioPharma Dive·6 months ago·Read at source →

Original title: “Lilly data point to ‘maintenance’ strategies for GLP-1 weight loss”

Research

The Case for Response Adaptive Trial Design for Rare Diseases | Applied Clinical Trials Online

Treatment & Clinical News·6 months ago·Read at source →

Research

The article emphasizes the need to accelerate rare disease research through innovative methodologies such as AI-powered in silico trials and digital twin-driven randomized controlled trials (RCTs). These approaches aim to enhance the quality of real-world evidence for decision-making in rare disease treatments.

time to accelerate rare disease research ai powered in silico trials digital twin driven rcts and decision grade real world evidence

PubMed Rare Disease·6 months ago·Read at source →

Original title: “Time to accelerate rare disease research: AI-powered in silico trials, digital twin-driven RCTs and decision-grade real-world evidence.”

Kabuki syndrome Kabuki syndrome 2+1 moreResearch

A new episignature for Kabuki syndrome enhances the understanding of KMT2D variants, paving the way for improved precision medicine approaches. This discovery could significantly impact patient management and treatment strategies.

next generation episignature for kabuki syndrome enables fine mapping of the impact of kmt2d variants to inform precision medicine

PubMed Rare Disease·6 months ago·Read at source →

Original title: “A next-generation episignature for Kabuki syndrome enables fine mapping of the impact of KMT2D variants to inform precision medicine.”

Research

A recent study published in PubMed demonstrates sustained remission in pediatric patients with bowel-associated dermatosis-arthritis syndrome following treatment with risankizumab. This finding highlights the potential of risankizumab as an effective therapy for this rare condition.

sustained remission of pediatric bowel associated dermatosis arthritis syndrome with risankizumab

PubMed Rare Disease·6 months ago·Read at source →

Original title: “Sustained Remission of Pediatric Bowel-Associated Dermatosis-Arthritis Syndrome With Risankizumab.”

Research

Development of novel 2-[(8-hydroxyquinolin-7-yl)(phenyl)methylamino]benzoic acid derivatives for inhibiting the catalytic activity of botulinum neurotoxins type B and F: in silico, in vitro & in vivo evaluation.

PubMed Rare Disease·6 months ago·Read at source →

VEXAS syndromeResearch

A case report details the first documented instance of VEXAS syndrome in Lebanon, highlighting the efficacy of Azacitidine as a therapeutic option. This adds to the understanding of VEXAS syndrome and potential treatment pathways.

first reported case of vexas syndrome in lebanon efficacy of azacitidine as a therapeutic option case report

PubMed Rare Disease·6 months ago·Read at source →

Original title: “The First Reported Case of VEXAS Syndrome in Lebanon: Efficacy of Azacitidine as a Therapeutic Option-Case Report.”

Research

A scoping review examines the impact of orthopedic surgery on activities of daily living and quality of life in patients with haemophilic arthropathy. This research highlights the need for improved postoperative care strategies to enhance patient outcomes.

activities of daily living and quality of life after orthopaedic surgery for haemophilic arthropathy a scoping review

PubMed Rare Disease·6 months ago·Read at source →

Original title: “Activities of Daily Living and Quality of Life After Orthopaedic Surgery for Haemophilic Arthropathy: A Scoping Review.”

resistant hypertension hyperaldosteronism+1 moreResearch

Recent research sheds light on the connections between resistant hypertension, hyperaldosteronism, and hypercortisolism, enhancing the understanding of these conditions. This emerging knowledge could inform future therapeutic strategies and patient management.

resistant hypertension hyperaldosteronism and the emerging understanding of hypercortisolism

PubMed Rare Disease·6 months ago·Read at source →

Original title: “Resistant hypertension, hyperaldosteronism, and the emerging understanding of hypercortisolism.”

myelofibrosisResearch

A study highlights the diagnostic challenges posed by the calreticulin type 26 mutation in myelofibrosis, a rare variant that complicates accurate diagnosis. Understanding this mutation is crucial for improving patient outcomes in myelofibrosis.

calreticulin type 26 mutation in myelofibrosis a rare variant with diagnostic challenges

PubMed Rare Disease·6 months ago·Read at source →

Original title: “Calreticulin Type 26 Mutation in Myelofibrosis: A Rare Variant With Diagnostic Challenges.”

methotrexate-associated lymphoproliferative disordersResearch

Recent research highlights how FAS-controlled T cells promote lymphoproliferation via glycolysis, bypassing typical effector differentiation. This discovery could inform future therapeutic strategies for related conditions.

fas controlled t cells drive lymphoproliferation through glycolysis without effector differentiation

PubMed Rare Disease·6 months ago·Read at source →

Original title: “FAS-controlled T cells drive lymphoproliferation through glycolysis without effector differentiation.”

aneuploidyResearch

A narrative review highlights maternal genetic variants linked to aneuploid conception, providing insights into potential genetic factors influencing reproductive outcomes. This research may inform future studies on reproductive health and genetic counseling.

maternal genetic variants associated with aneuploid conception a narrative review

PubMed Rare Disease·6 months ago·Read at source →

Original title: “Maternal genetic variants associated with aneuploid conception: a narrative review.”

thymic neuroendocrine tumor pancreatic insulin-producing neuroendocrine tumor+1 moreResearch

A recent study published in PubMed highlights the clinicopathological features and outcomes of immature PIT-1 lineage tumors, identified as a high-risk subtype of pituitary neuroendocrine tumors according to WHO 2022. This research provides valuable insights into the characteristics and prognosis of this rare tumor type.

clinicopathological features and outcomes of immature pit 1 lineage tumors a high risk pituitary neuroendocrine tumor pituitary adenoma subtype per who 2022

PubMed Rare Disease·6 months ago·Read at source →

Original title: “Clinicopathological features and outcomes of immature PIT-1 lineage tumors: A high-risk pituitary neuroendocrine tumor/pituitary adenoma subtype per WHO 2022.”

chronic inflammatory demyelinating polyradiculoneuropathy polyneuropathy, inflammatory demyelinating, chronicResearch

A corrigendum has been published regarding the diagnostic adjudication of potential participants with chronic inflammatory demyelinating polyradiculoneuropathy in the ADHERE trial of subcutaneous efgartigimod PH20. This update addresses previous findings in the Journal of the Neurological Sciences.

corrigendum to diagnostic adjudication of potential participants with chronic inflammatory demyelinating polyradiculoneuropathy in the adhere trial of subcutaneous efgartigimod ph20 journal of the neurological sciences 486 2026 125918

PubMed Rare Disease·6 months ago·Read at source →

Original title: “Corrigendum to 'Diagnostic adjudication of potential participants with chronic inflammatory demyelinating polyradiculoneuropathy in the ADHERE trial of subcutaneous efgartigimod PH20' [Journal of the Neurological Sciences, 486 (2026) 125918].”

class V glucose-6-phosphate dehydrogenase deficiency G6PD deficiency+1 moreResearch

A case report highlights profound thrombocytopenia associated with tirofiban in a patient with glucose-6-phosphate dehydrogenase deficiency during primary percutaneous coronary intervention. This finding underscores the need for caution when using tirofiban in patients with this genetic deficiency.

tirofiban associated profound thrombocytopenia in a patient with glucose 6 phosphate dehydrogenase deficiency undergoing primary percutaneous coronary intervention a case report

PubMed Rare Disease·6 months ago·Read at source →

Original title: “Tirofiban-Associated Profound Thrombocytopenia in a Patient With Glucose-6-Phosphate Dehydrogenase Deficiency Undergoing Primary Percutaneous Coronary Intervention: A Case Report.”

brachial plexus neuritis brachial plexus neuropathy+1 moreResearch

A recent study published in PubMed discusses the surgical management of pediatric brachial plexus lipoma, highlighting techniques and outcomes. This research contributes to the understanding of treatment options for this rare condition.

surgical management of a paediatric brachial plexus lipoma

PubMed Rare Disease·6 months ago·Read at source →

Original title: “Surgical management of a paediatric brachial plexus lipoma.”

Research

A new study discusses the repair of a unicuspid aortic valve using root stabilization techniques. This research contributes to the understanding of surgical options for patients with this specific heart valve anomaly.

repair of a unicuspid aortic valve with root stabilization

PubMed Rare Disease·6 months ago·Read at source →

Original title: “Repair of a unicuspid aortic valve with root stabilization.”

Research

Research indicates that semaglutide reduces neuroinflammation and cognitive impairment in APP/PS1 mouse models, suggesting potential therapeutic effects for Alzheimer's disease. This study contributes to the understanding of semaglutide's broader applications beyond diabetes management.

semaglutide ameliorates neuroinflammation and cognitive impairment in app ps1 mice

PubMed Rare Disease·6 months ago·Read at source →

Original title: “Semaglutide ameliorates neuroinflammation and cognitive impairment in APP/PS1 mice.”

Rasmussen subacute encephalitisResearch

A recent study published in PubMed explores the immune landscape of the brain in patients with Rasmussen encephalitis. This research provides insights into the immune mechanisms involved in this rare neurological disorder.

immune landscape of the affected brain in rasmussen encephalitis

PubMed Rare Disease·6 months ago·Read at source →

Original title: “Immune landscape of the affected brain in Rasmussen encephalitis.”

Previous307 / 487Next

Daily Briefings

Not rare diseases news,news about your rare disease.

Earlier

Analysis of Brain Tumor Following AAV Gene Therapy Identifies Vector Integration and Reinforces Importance of Long-Term Monitoring | Children's Hospital of Philadelphia

Recent trial results indicate that transitioning from high-dose GLP-1 injectables to lower doses or oral formulations may enhance long-term weight maintenance in individuals with obesity. These findings could influence future treatment strategies in obesity management.

The Case for Response Adaptive Trial Design for Rare Diseases | Applied Clinical Trials Online

A new episignature for Kabuki syndrome enhances the understanding of KMT2D variants, paving the way for improved precision medicine approaches. This discovery could significantly impact patient management and treatment strategies.

A recent study published in PubMed demonstrates sustained remission in pediatric patients with bowel-associated dermatosis-arthritis syndrome following treatment with risankizumab. This finding highlights the potential of risankizumab as an effective therapy for this rare condition.

Development of novel 2-[(8-hydroxyquinolin-7-yl)(phenyl)methylamino]benzoic acid derivatives for inhibiting the catalytic activity of botulinum neurotoxins type B and F: in silico, in vitro & in vivo evaluation.

A case report details the first documented instance of VEXAS syndrome in Lebanon, highlighting the efficacy of Azacitidine as a therapeutic option. This adds to the understanding of VEXAS syndrome and potential treatment pathways.

A scoping review examines the impact of orthopedic surgery on activities of daily living and quality of life in patients with haemophilic arthropathy. This research highlights the need for improved postoperative care strategies to enhance patient outcomes.

Recent research sheds light on the connections between resistant hypertension, hyperaldosteronism, and hypercortisolism, enhancing the understanding of these conditions. This emerging knowledge could inform future therapeutic strategies and patient management.

A study highlights the diagnostic challenges posed by the calreticulin type 26 mutation in myelofibrosis, a rare variant that complicates accurate diagnosis. Understanding this mutation is crucial for improving patient outcomes in myelofibrosis.

Recent research highlights how FAS-controlled T cells promote lymphoproliferation via glycolysis, bypassing typical effector differentiation. This discovery could inform future therapeutic strategies for related conditions.

A narrative review highlights maternal genetic variants linked to aneuploid conception, providing insights into potential genetic factors influencing reproductive outcomes. This research may inform future studies on reproductive health and genetic counseling.

A corrigendum has been published regarding the diagnostic adjudication of potential participants with chronic inflammatory demyelinating polyradiculoneuropathy in the ADHERE trial of subcutaneous efgartigimod PH20. This update addresses previous findings in the Journal of the Neurological Sciences.

A case report highlights profound thrombocytopenia associated with tirofiban in a patient with glucose-6-phosphate dehydrogenase deficiency during primary percutaneous coronary intervention. This finding underscores the need for caution when using tirofiban in patients with this genetic deficiency.

A recent study published in PubMed discusses the surgical management of pediatric brachial plexus lipoma, highlighting techniques and outcomes. This research contributes to the understanding of treatment options for this rare condition.

A new study discusses the repair of a unicuspid aortic valve using root stabilization techniques. This research contributes to the understanding of surgical options for patients with this specific heart valve anomaly.

Research indicates that semaglutide reduces neuroinflammation and cognitive impairment in APP/PS1 mouse models, suggesting potential therapeutic effects for Alzheimer's disease. This study contributes to the understanding of semaglutide's broader applications beyond diabetes management.

A recent study published in PubMed explores the immune landscape of the brain in patients with Rasmussen encephalitis. This research provides insights into the immune mechanisms involved in this rare neurological disorder.

Not rare diseases news,
news about your rare disease.