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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

Liver Gene Therapy in Fabry Disease Mice With Low Doses of rAAV2/8 Expressing a Codon-Optimized hGLA cDNA Results in Long-Term Disease Correction.

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

Research

Rejuvenate crowdfunds development of gene therapy for aging, chronic diseases

BioSpace·7 months ago·Read at source →

neonatal abstinence syndrome neonatal antiphospholipid syndromeResearch

A NIH-funded clinical trial demonstrates that a symptom-based approach effectively treats opioid withdrawal in newborns with minimal drug exposure, potentially reducing hospital stay durations. This innovative strategy could reshape care protocols for affected infants.

NIH-funded clinical trial shows that the new approach could also decrease time in the hospital.

NIH News·7 months ago·Read at source →

Original title: “Symptom-based approach treats opioid withdrawal in newborns with minimal drug exposure ”

Gaucher disease type I Gaucher disease+1 moreResearch

A recent study highlights left ventricular global longitudinal strain as a potential early predictor of disease severity progression in Gaucher disease type 1. This finding could enhance monitoring and treatment strategies for affected patients.

left ventricular global longitudinal strain as an early predictor of disease severity progression in gaucher disease type 1

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

Original title: “Left Ventricular Global Longitudinal Strain as an Early Predictor of Disease Severity Progression in Gaucher Disease Type 1.”

Niemann-Pick disease type CResearch

New consensus clinical management guidelines for Niemann-Pick Disease Type C have been published, aiming to standardize care and improve patient outcomes. These guidelines are crucial for healthcare providers managing this rare condition.

2025 consensus clinical management guidelines for niemann pick disease type c

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

Original title: “2025 Consensus Clinical Management Guidelines for Niemann-Pick Disease Type C.”

calciphylaxis calciphylaxis cutis+1 moreResearch

A case study highlights the diagnostic challenges of early calciphylaxis in a patient with end-stage renal disease and sarcoidosis. The findings emphasize the need for careful wound management and awareness of calciphylaxis symptoms in similar patients.

early calciphylaxis mimicking infection in a patient with end stage renal disease and sarcoidosis a diagnostic and wound management challenge

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

Original title: “Early Calciphylaxis Mimicking Infection in a Patient With End-Stage Renal Disease and Sarcoidosis: A Diagnostic and Wound Management Challenge.”

postpartum psychosis alcoholic psychosis+1 moreResearch

A recent study explores the clinical management of psychosis in a 47,XXX adolescent, highlighting the importance of a multidisciplinary approach. This case contributes to the understanding of psychiatric manifestations associated with chromosomal abnormalities.

psychosis in a 47 xxx adolescent clinical management and multidisciplinary follow up

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

Original title: “Psychosis in a 47,XXX Adolescent: Clinical Management and Multidisciplinary Follow-up.”

Research

Gene therapy leaders aim to help more Baby KJs with novel regulatory models

BioSpace·7 months ago·Read at source →

Yoon-Bellen neurodevelopmental syndrome Brunet-Wagner neurodevelopmental syndromeResearch

Research identifies a severe neurodevelopmental syndrome associated with a South Asian founder variant in the UFMylation adaptor CDK5RAP3. This discovery enhances understanding of the genetic underpinnings of neurodevelopmental disorders.

severe neurodevelopmental syndrome linked to a south asian founder variant in the ufmylation adaptor cdk5rap3

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

Original title: “A severe neurodevelopmental syndrome linked to a South Asian founder variant in the UFMylation adaptor CDK5RAP3.”

arginase deficiencyResearch

A novel hybrid piggyBac AAV-transposon and LNP-transposase system demonstrates a 100-fold dose reduction for treating arginase deficiency compared to traditional AAV methods. This breakthrough could enhance treatment efficacy and reduce costs for patients.

hybrid piggybac aav transposon and lnp transposase system for treating arginase deficiency a 100 fold dose reduction compared to aav alone

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

Original title: “A Hybrid PiggyBac AAV-Transposon and LNP-Transposase System for Treating Arginase Deficiency: A 100-fold Dose Reduction Compared to AAV Alone.”

Research

A recent randomized clinical trial has evaluated the efficacy and safety of vemircopan in patients with generalized myasthenia gravis. The findings contribute valuable insights into potential treatment options for this rare neuromuscular disorder.

efficacy and safety of vemircopan in generalized myasthenia gravis a randomized clinical trial

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

Original title: “Efficacy and Safety of Vemircopan in Generalized Myasthenia Gravis: A Randomized Clinical Trial.”

Nijmegen breakage syndrome Nijmegen breakage syndrome-like disorderResearch

Derivation of homozygous Nijmegen breakage syndrome induced pluripotent stem cell line (MUNIi019-A).

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

Research

Urine-Derived Stem Cells: Applications of Paracrine-Driven Regeneration and Functional Recovery in Multiorgan Systems.

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

Research

A recent study explores the role of the gut microbiome in pediatric-onset acquired demyelinating syndromes, focusing on myelin oligodendrocyte glycoprotein antibody status. This research may provide insights into potential therapeutic targets for these conditions.

gut microbiome in pediatric onset acquired demyelinating syndromes by myelin oligodendrocyte glycoprotein antibody status

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

Original title: “The gut microbiome in pediatric-onset acquired demyelinating syndromes by myelin oligodendrocyte glycoprotein antibody status.”

Research

A recent study elucidates the diagnostic challenges of atypical long-chain 3-hydroxyacyl-CoA dehydrogenase deficiency (LCHADD) by identifying three allelic products resulting from two pathogenic variants. This research enhances understanding of the genetic underpinnings of LCHADD, potentially guiding future diagnostic approaches.

diagnostic odyssey of atypical long chain 3 hydroxyacyl coa dehydrogenase deficiency lchadd explained by three allelic products from two pathogenic variants

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

Original title: “Diagnostic Odyssey of Atypical Long-Chain 3-Hydroxyacyl-CoA Dehydrogenase Deficiency (LCHADD) Explained by Three Allelic Products From Two Pathogenic Variants.”

congenital candidiasisResearch

A case study highlights the successful treatment of severe congenital candidiasis caused by Candida dubliniensis in a preterm infant using fosfluconazole. This research contributes to understanding treatment options for this rare fungal infection.

severe congenital candidiasis due to candida dubliniensis treated with fosfluconazole in a preterm infant

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

Original title: “Severe Congenital Candidiasis due to Candida dubliniensis Treated With Fosfluconazole in a Preterm Infant.”

necrotizing enterocolitis perinatal necrotizing enterocolitisResearch

Recent research highlights the role of the SUCNR1/HIF-1α/BNIP3 axis in exacerbating necrotizing enterocolitis through succinate-induced apoptosis in intestinal epithelial cells. This discovery could inform future therapeutic strategies for this serious condition.

necrotizing enterocolitis is exacerbated through sucnr1 hif 1 bnip3 axis mediated succinate induced intestinal epithelial cell apoptosis

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

Original title: “Necrotizing enterocolitis is exacerbated through SUCNR1/HIF-1α/BNIP3 axis-mediated succinate-induced intestinal epithelial cell apoptosis.”

achalasia idiopathic achalasia+1 moreResearch

Recent research highlights advancements in endoscopic and surgical therapies for achalasia, a rare esophageal disorder. These modern treatment options aim to improve patient outcomes and quality of life.

modern treatment for achalasia endoscopic and surgical therapies

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

Original title: “Modern Treatment for Achalasia: Endoscopic and Surgical therapies.”

inclusion body myositisResearch

A new study explores the use of muscle ultrasound in inclusion body myositis, integrating qualitative and quantitative methods alongside clinical and MRI findings. This research may enhance diagnostic accuracy and patient management strategies.

muscle ultrasound in inclusion body myositis integrating qualitative and quantitative approaches with clinical and mri findings

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

Original title: “Muscle Ultrasound in Inclusion Body Myositis: Integrating Qualitative and Quantitative Approaches With Clinical and MRI Findings.”

Research

A rare case report details synovial lipomatosis of the ankle associated with sural nerve neuropathy. This case contributes to the understanding of the clinical implications of this uncommon condition.

synovial lipomatosis of ankle with sural nerve neuropathy a rare case report

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

Original title: “Synovial Lipomatosis of Ankle with Sural Nerve Neuropathy: A Rare Case Report.”

Previous341 / 488Next

Daily Briefings

Not rare diseases news,news about your rare disease.

Earlier

Liver Gene Therapy in Fabry Disease Mice With Low Doses of rAAV2/8 Expressing a Codon-Optimized hGLA cDNA Results in Long-Term Disease Correction.

Rejuvenate crowdfunds development of gene therapy for aging, chronic diseases

A NIH-funded clinical trial demonstrates that a symptom-based approach effectively treats opioid withdrawal in newborns with minimal drug exposure, potentially reducing hospital stay durations. This innovative strategy could reshape care protocols for affected infants.

A recent study highlights left ventricular global longitudinal strain as a potential early predictor of disease severity progression in Gaucher disease type 1. This finding could enhance monitoring and treatment strategies for affected patients.

New consensus clinical management guidelines for Niemann-Pick Disease Type C have been published, aiming to standardize care and improve patient outcomes. These guidelines are crucial for healthcare providers managing this rare condition.

A case study highlights the diagnostic challenges of early calciphylaxis in a patient with end-stage renal disease and sarcoidosis. The findings emphasize the need for careful wound management and awareness of calciphylaxis symptoms in similar patients.

A recent study explores the clinical management of psychosis in a 47,XXX adolescent, highlighting the importance of a multidisciplinary approach. This case contributes to the understanding of psychiatric manifestations associated with chromosomal abnormalities.

Gene therapy leaders aim to help more Baby KJs with novel regulatory models

Research identifies a severe neurodevelopmental syndrome associated with a South Asian founder variant in the UFMylation adaptor CDK5RAP3. This discovery enhances understanding of the genetic underpinnings of neurodevelopmental disorders.

A novel hybrid piggyBac AAV-transposon and LNP-transposase system demonstrates a 100-fold dose reduction for treating arginase deficiency compared to traditional AAV methods. This breakthrough could enhance treatment efficacy and reduce costs for patients.

A recent randomized clinical trial has evaluated the efficacy and safety of vemircopan in patients with generalized myasthenia gravis. The findings contribute valuable insights into potential treatment options for this rare neuromuscular disorder.

Derivation of homozygous Nijmegen breakage syndrome induced pluripotent stem cell line (MUNIi019-A).

Urine-Derived Stem Cells: Applications of Paracrine-Driven Regeneration and Functional Recovery in Multiorgan Systems.

A recent study explores the role of the gut microbiome in pediatric-onset acquired demyelinating syndromes, focusing on myelin oligodendrocyte glycoprotein antibody status. This research may provide insights into potential therapeutic targets for these conditions.

A case study highlights the successful treatment of severe congenital candidiasis caused by Candida dubliniensis in a preterm infant using fosfluconazole. This research contributes to understanding treatment options for this rare fungal infection.

Recent research highlights the role of the SUCNR1/HIF-1α/BNIP3 axis in exacerbating necrotizing enterocolitis through succinate-induced apoptosis in intestinal epithelial cells. This discovery could inform future therapeutic strategies for this serious condition.

Recent research highlights advancements in endoscopic and surgical therapies for achalasia, a rare esophageal disorder. These modern treatment options aim to improve patient outcomes and quality of life.

A new study explores the use of muscle ultrasound in inclusion body myositis, integrating qualitative and quantitative methods alongside clinical and MRI findings. This research may enhance diagnostic accuracy and patient management strategies.

A rare case report details synovial lipomatosis of the ankle associated with sural nerve neuropathy. This case contributes to the understanding of the clinical implications of this uncommon condition.

Not rare diseases news,
news about your rare disease.