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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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myocarditis acute myocarditis+1 moreResearch

A recent study details a case of fulminant recurrent myocarditis complicated by papillary muscle rupture and cardiogenic shock, successfully managed with extracorporeal membrane oxygenation and Impella support. This case highlights innovative approaches in critical cardiac care.

fulminant recurrent myocarditis complicated by papillary muscle rupture and cardiogenic shock managed with extracorporeal membrane oxygenation and impella support

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

Original title: “Fulminant recurrent myocarditis complicated by papillary muscle rupture and cardiogenic shock managed with ExtraCorporeal membrane oxygenation and impella support.”

Research

A new publication outlines ten proposals aimed at enhancing regulatory frameworks for drug repurposing, focusing on improving access and safety for patients. These recommendations could streamline processes and foster innovation in treating rare diseases.

regulatory innovation for drug repurposing ten proposals to accelerate access and safety

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

Original title: “Regulatory innovation for drug repurposing: Ten proposals to accelerate access and safety.”

hidradenitis suppurativaResearch

Recent research explores the genetic underpinnings of hidradenitis suppurativa, shedding light on potential biomarkers and therapeutic targets. This study contributes to the understanding of the disease's etiology and may inform future treatment strategies.

genetics of hidradenitis suppurativa

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

Original title: “The Genetics of Hidradenitis Suppurativa.”

adrenocortical carcinoma, hereditaryResearch

A case study highlights non-functioning adrenocortical carcinoma presenting with acute spontaneous retroperitoneal hemorrhage. This discovery may enhance understanding of the disease's clinical manifestations.

non functioning adrenocortical carcinoma presenting as acute spontaneous retroperitoneal haemorrhage

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

Original title: “Non-functioning adrenocortical carcinoma presenting as acute spontaneous retroperitoneal haemorrhage.”

asthmaResearch

A two-year follow-up study reveals that dupilumab can induce eosinophilia in patients with severe asthma, both in biologic-naïve and previously treated groups. This real-life evidence contributes to understanding the long-term effects of dupilumab in asthma management.

dupilumab induced eosinophilia in severe asthma 2 year follow up real life evidence from biologic na ve and previously treated patients

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

Original title: “Dupilumab-induced eosinophilia in severe asthma: 2-year follow-up real-life evidence from biologic-naïve and previously treated patients.”

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

A recent study evaluates the lifetime costs associated with severe visual impairment due to retinitis pigmentosa. This research highlights the economic burden of the disease, providing valuable insights for stakeholders in healthcare and policy.

assessment of the lifetime costs of severe visual impairment due to retinitis pigmentosa

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

Original title: “Assessment of the Lifetime Costs of Severe Visual Impairment Due to Retinitis Pigmentosa.”

major depressive disorder major depressive disorder 1+1 moreResearch

The international PROMPT study reveals that childhood trauma significantly impacts the severity of depressive symptoms and drug resistance in major depressive disorder. These findings could inform future treatment strategies for affected individuals.

effect of childhood trauma on the severity of depressive symptoms and drug resistance in major depressive disorder results from the international prompt study

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

Original title: “The effect of childhood trauma on the severity of depressive symptoms and drug resistance in major depressive disorder - results from the international PROMPT study.”

Research

A recent study explores the application of artificial intelligence algorithms in diagnosing challenging cases, highlighting the gap between potential benefits and actual clinical support. This research could influence future diagnostic practices in rare diseases.

role of artificial intelligence algorithms in challenging diagnostic cases between potential and real clinical support

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

Original title: “The Role of Artificial Intelligence Algorithms in Challenging Diagnostic Cases - Between Potential and Real Clinical Support.”

Research

A new study characterizes structural and functional alterations in ADSS1-deficient myopathy using multimodal noninvasive biomarkers. This research could enhance understanding and diagnosis of this rare muscle disorder.

multimodal noninvasive biomarker characterization of structural and functional alterations in adss1 deficient myopathy

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

Original title: “Multimodal Noninvasive Biomarker Characterization of Structural and Functional Alterations in ADSS1-Deficient Myopathy.”

obesity disorderResearch

Roche and Zealand Pharma report that their amylin analog petrelintide achieved a 9% weight reduction in a placebo-controlled study over 42 weeks. This result, however, did not meet the expectations of analysts and investors.

Roche and Zealand Pharma announced last month that their amylin analog petrelintide elicited a 9% placebo-controlled weight reduction at 42 weeks—falling far below analyst and investor expectations.

BioSpace·7 months ago·Read at source →

Original title: “Roche insists amylin obesity drug still valuable for patients who ‘don’t want side effects’”

myasthenia gravis transient neonatal myasthenia gravisResearch

At the AAN 2026 meeting, Johnson & Johnson, Kyverna, Capricor, and Praxis presented data that could redefine treatment standards for myasthenia gravis. These findings may influence future clinical practices and therapeutic approaches in the field.

This year’s American Academy of Neurology meeting included a presentation that could one day set a new treatment standard for myasthenia gravis.

BioSpace·7 months ago·Read at source →

Original title: “AAN 2026: J&J, Kyverna, Capricor and Praxis showcase practice-changing data”

Research

Research highlights that depletion of the TECPR2 gene results in mitochondrial failure linked to neurodegeneration. This discovery may provide insights into potential therapeutic targets for related neurodegenerative diseases.

depletion of tecpr2 leads to mitochondrial failure associated with neurodegeneration

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

Original title: “Depletion of TECPR2 leads to mitochondrial failure associated with neurodegeneration.”

Research

Prenylated acylphloroglucinols from the fruits of Hypericum patulum.

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

Research

Radiological complete response in diffuse intrinsic pontine glioma: a case report.

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

Research

A recent study highlights the predictive value of peripheral blood cell parameters and their ratios in determining intravenous immunoglobulin resistance in Kawasaki disease. This research could enhance patient management strategies for this condition.

value of peripheral blood cell parameters and their derived ratios in predicting intravenous immunoglobulin resistance in kawasaki disease

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

Original title: “Value of peripheral blood cell parameters and their derived ratios in predicting intravenous immunoglobulin resistance in Kawasaki disease.”

recessive dystrophic epidermolysis bullosa recessive dystrophic epidermolysis bullosa-generalized other+1 moreResearch

A case report highlights the challenges of managing recessive dystrophic epidermolysis bullosa in a resource-limited setting at Mohamed Adam Sheikh Children's Teaching Hospital in Hargeisa, Somaliland. This study underscores the need for improved healthcare resources for rare diseases in underserved regions.

recessive dystrophic epidermolysis bullosa in a resource limited setting a case report from mohamed adam sheikh children s teaching hospital hargeisa somaliland

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

Original title: “Recessive Dystrophic Epidermolysis Bullosa in a Resource-Limited Setting: A Case Report From Mohamed Adam Sheikh Children's Teaching Hospital Hargeisa, Somaliland.”

Research

A retrospective study highlights the predominance of in-field recurrence after radiotherapy in sinonasal cancer patients. This research underscores the need for improved treatment strategies to enhance patient outcomes.

predominance of in field recurrence after radiotherapy for sinonasal cancer a single center retrospective study

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

Original title: “Predominance of In-Field Recurrence After Radiotherapy for Sinonasal Cancer: A Single-Center Retrospective Study.”

Research

Research identifies aldolase and pyruvate kinase as new allergens from cockroaches, specifically Per a 21 and Per a 22. This discovery could enhance understanding of allergic responses related to cockroach exposure.

identification of aldolase and pyruvate kinase as novel cockroach allergens per a 21 and per a 22

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

Original title: “Identification of aldolase and pyruvate kinase as novel cockroach allergens, Per a 21 and Per a 22.”

Lynch syndrome Lynch syndrome 1+1 moreResearch

A case report highlights immune checkpoint inhibitor resistance in a patient with high-grade intraepithelial neoplasia associated with Lynch syndrome and colon cancer. This study contributes to understanding treatment challenges in dMMR tumors.

immune checkpoint inhibitor resistance in dmmr high grade intraepithelial neoplasia with lynch syndrome associated colon cancer a case report

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

Original title: “Immune checkpoint inhibitor resistance in dMMR high-grade intraepithelial neoplasia with lynch syndrome-associated colon cancer: a case report.”

coccidioidomycosisResearch

A recent study explores neonatal management and outcomes for infants born to mothers with disseminated coccidioidomycosis during pregnancy. The findings contribute to understanding the implications of this rare fungal infection on neonatal health.

neonatal management and outcomes in the setting of disseminated coccidioidomycosis in pregnancy

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

Original title: “Neonatal management and outcomes in the setting of disseminated coccidioidomycosis in pregnancy.”

Previous348 / 488Next

Daily Briefings

Not rare diseases news,news about your rare disease.

Earlier

A recent study details a case of fulminant recurrent myocarditis complicated by papillary muscle rupture and cardiogenic shock, successfully managed with extracorporeal membrane oxygenation and Impella support. This case highlights innovative approaches in critical cardiac care.

A new publication outlines ten proposals aimed at enhancing regulatory frameworks for drug repurposing, focusing on improving access and safety for patients. These recommendations could streamline processes and foster innovation in treating rare diseases.

Recent research explores the genetic underpinnings of hidradenitis suppurativa, shedding light on potential biomarkers and therapeutic targets. This study contributes to the understanding of the disease's etiology and may inform future treatment strategies.

A case study highlights non-functioning adrenocortical carcinoma presenting with acute spontaneous retroperitoneal hemorrhage. This discovery may enhance understanding of the disease's clinical manifestations.

A two-year follow-up study reveals that dupilumab can induce eosinophilia in patients with severe asthma, both in biologic-naïve and previously treated groups. This real-life evidence contributes to understanding the long-term effects of dupilumab in asthma management.

A recent study evaluates the lifetime costs associated with severe visual impairment due to retinitis pigmentosa. This research highlights the economic burden of the disease, providing valuable insights for stakeholders in healthcare and policy.

The international PROMPT study reveals that childhood trauma significantly impacts the severity of depressive symptoms and drug resistance in major depressive disorder. These findings could inform future treatment strategies for affected individuals.

A recent study explores the application of artificial intelligence algorithms in diagnosing challenging cases, highlighting the gap between potential benefits and actual clinical support. This research could influence future diagnostic practices in rare diseases.

A new study characterizes structural and functional alterations in ADSS1-deficient myopathy using multimodal noninvasive biomarkers. This research could enhance understanding and diagnosis of this rare muscle disorder.

Roche and Zealand Pharma report that their amylin analog petrelintide achieved a 9% weight reduction in a placebo-controlled study over 42 weeks. This result, however, did not meet the expectations of analysts and investors.

At the AAN 2026 meeting, Johnson & Johnson, Kyverna, Capricor, and Praxis presented data that could redefine treatment standards for myasthenia gravis. These findings may influence future clinical practices and therapeutic approaches in the field.

Research highlights that depletion of the TECPR2 gene results in mitochondrial failure linked to neurodegeneration. This discovery may provide insights into potential therapeutic targets for related neurodegenerative diseases.

Prenylated acylphloroglucinols from the fruits of Hypericum patulum.

Radiological complete response in diffuse intrinsic pontine glioma: a case report.

A recent study highlights the predictive value of peripheral blood cell parameters and their ratios in determining intravenous immunoglobulin resistance in Kawasaki disease. This research could enhance patient management strategies for this condition.

A retrospective study highlights the predominance of in-field recurrence after radiotherapy in sinonasal cancer patients. This research underscores the need for improved treatment strategies to enhance patient outcomes.

Research identifies aldolase and pyruvate kinase as new allergens from cockroaches, specifically Per a 21 and Per a 22. This discovery could enhance understanding of allergic responses related to cockroach exposure.

A case report highlights immune checkpoint inhibitor resistance in a patient with high-grade intraepithelial neoplasia associated with Lynch syndrome and colon cancer. This study contributes to understanding treatment challenges in dMMR tumors.

A recent study explores neonatal management and outcomes for infants born to mothers with disseminated coccidioidomycosis during pregnancy. The findings contribute to understanding the implications of this rare fungal infection on neonatal health.

Not rare diseases news,
news about your rare disease.