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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.

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

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Research

FDA Draft Guidance Could Streamline Rare Disease Gene Therapy Development | BioPharm International

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

Kleefstra syndrome 1 Abruzzo-Erickson syndrome+1 moreResearch

EHMT1 Gene Therapy for Kleefstra Syndrome Enters Preclinical Collaboration | BioPharm International

Treatment & Clinical News·5 months ago·Read at source →

Research

Institute for Pediatric Rare Diseases at FSU, A.J. Anderson Foundation expand access to genetic screening and rare disease care - Florida State University News

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

Research

Pelage Pharmaceuticals and Eli Lilly discuss innovative approaches to hair loss treatment, focusing on regenerative methods to reactivate dormant hair follicles. Early proof-of-concept findings could signal a shift in the long-stagnant hair loss treatment market.

In this episode of Denatured, you'll be hearing from Daniel Gil, CEO of Pelage Pharmaceuticals and Francisco Ramírez-Valle, senior vice president of immunology discovery at Eli Lilly. We dive into the long-overlooked hair loss space, exploring why true innovation has lagged, how a regenerative approach aims to reactivate dormant follicles and what early proof-of-concept means for patients.

BioSpace·5 months ago·Read at source →

Original title: “Rethinking hair loss treatment”

large cell neuroendocrine carcinoma pancreatic large cell neuroendocrine carcinoma+1 moreResearch

A case report and literature review on primary large cell neuroendocrine carcinoma of the pulmonary artery has been published, contributing to the understanding of this rare cancer type. The findings may inform future research and clinical approaches.

primary large cell neuroendocrine carcinoma of pulmonary artery a case report and literature review

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

Original title: “[Primary large cell neuroendocrine carcinoma of pulmonary artery: a case report and literature review].”

autoimmune pulmonary alveolar proteinosis hereditary pulmonary alveolar proteinosisResearch

A case report highlights autoimmune pulmonary alveolar proteinosis induced by brigatinib, contributing to the understanding of drug-related adverse effects. This literature review may inform clinicians about potential risks associated with brigatinib treatment.

autoimmune pulmonary alveolar proteinosis induced by brigatinib a case report and literature review

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

Original title: “[Autoimmune pulmonary alveolar proteinosis induced by brigatinib: a case report and literature review].”

Research

A new study explores the pathogenesis of fibrosing mediastinitis, shedding light on common etiologies associated with this rare condition. Understanding these mechanisms may inform future therapeutic strategies.

pathogenesis of fibrosing mediastinitis caused by common etiologies

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

Original title: “[Pathogenesis of fibrosing mediastinitis caused by common etiologies].”

Kleefstra syndrome Kleefstra syndrome 1+1 moreResearch

IDefine and UT Southwestern Announce Research Collaboration to Advance Gene Therapy for Rare Disease Kleefstra Syndrome - BioSpace

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

Research

A recent study reveals that 21 orofacial diseases share a common genetic architecture with systemic diseases, highlighting potential genetic links. This integrative genomic analysis could pave the way for new therapeutic strategies and insights into disease mechanisms.

integrative genomic analysis of 21 orofacial diseases identifies shared genetic architecture with systemic diseases

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

Original title: “Integrative genomic analysis of 21 orofacial diseases identifies shared genetic architecture with systemic diseases.”

coronary artery disease, autosomal dominant 2 coronary artery disease, autosomal dominant, 1Research

A recent study utilizes machine learning to identify rare genetic variants that contribute to coronary artery disease risk in the Japanese population. This research enhances predictive capabilities, potentially guiding future genetic screening and intervention strategies.

machine learning reveals the contribution of rare genetic variants and enhances risk prediction for coronary artery disease in the japanese population

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

Original title: “Machine Learning Reveals the Contribution of Rare Genetic Variants and Enhances Risk Prediction for Coronary Artery Disease in the Japanese Population.”

Research

AAV gene therapy for homozygous familial hypercholesterolemia: a phase 1 trial.

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

Research

A new study expands the understanding of post-streptococcal neutrophilic pustular dermatosis, contributing to the histopathological framework of reactive neutrophilic dermatoses. This research may enhance diagnostic accuracy and treatment approaches for affected patients.

post streptococcal neutrophilic pustular dermatosis expanding the histopathological paradigm of reactive neutrophilic dermatoses

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

Original title: “Post-Streptococcal Neutrophilic Pustular Dermatosis: Expanding the Histopathological Paradigm of Reactive Neutrophilic Dermatoses.”

Research

A case report details triple-vessel coronary involvement and a giant aneurysm in a 6-year-old boy with Kawasaki disease. This study highlights the severe cardiovascular complications that can arise from Kawasaki disease, emphasizing the need for early detection and management.

triple vessel coronary involvement and giant aneurysm in a 6 year old boy with kawasaki disease a case report

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

Original title: “Triple-vessel coronary involvement and giant aneurysm in a 6-year-old boy with kawasaki disease: a case report.”

Turner syndrome Wilson-Turner syndromeResearch

A rare case report details the co-occurrence of moyamoya syndrome and Turner syndrome in a 4-year-old girl from Sudan. This case highlights the complexities of diagnosing and managing multiple rare conditions simultaneously.

co occurrence of moyamoya syndrome and turner syndrome in a 4 year old sudanese girl a rare case report

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

Original title: “Co-occurrence of moyamoya syndrome and Turner syndrome in a 4-year-old Sudanese girl: a rare case report.”

steroid inherited metabolic disorderResearch

A recent study focuses on optimizing formulations of Levocarnitine for pediatric patients with inherited metabolic disorders. This research highlights the importance of tailored treatments to improve therapeutic outcomes in this vulnerable population.

optimising formulations for paediatric patients with inherited metabolic disorders the case study of levocarnitine

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

Original title: “Optimising Formulations for Paediatric Patients with Inherited Metabolic Disorders: The Case Study of Levocarnitine.”

sarcoidosisResearch

A comprehensive review highlights granulomatous diseases that can mimic sarcoidosis, providing insights into differential diagnosis. This research may aid clinicians in recognizing and managing these conditions more effectively.

sarcoid like reactions a comprehensive review of granulomatous diseases mimicking sarcoidosis

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

Original title: “Sarcoid-like reactions: a comprehensive review of granulomatous diseases mimicking sarcoidosis.”

Research

A new study reports the first identification of the BRAF V600E mutation in paratesticular mesothelioma, potentially opening avenues for targeted therapies. This discovery highlights the genetic underpinnings of this rare cancer type.

first report of braf v600e mutation in paratesticular mesothelioma

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

Original title: “The First Report of BRAF V600E Mutation in Paratesticular Mesothelioma.”

Research

A pictorial review highlights interlobular septal thickening, providing insights into its radiological features. This review may aid in the diagnosis and understanding of related pulmonary conditions.

interlobular septal thickening a pictorial review

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

Original title: “Interlobular Septal Thickening: A Pictorial Review.”

respiratory syncytial virus infectious disease respiratory syncytial virus bronchiolitisResearch

A 20-year pharmacovigilance study evaluates the safety profile of palivizumab, revealing insights from the FDA Adverse Event Reporting System. This research contributes to understanding the long-term effects of the drug in treating respiratory syncytial virus.

post marketing safety profile of palivizumab a 20 year pharmacovigilance study using the fda adverse event reporting system faers

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

Original title: “Post-marketing safety profile of palivizumab: a 20-year pharmacovigilance study using the FDA Adverse Event Reporting System (FAERS).”

atherosclerotic cardiovascular diseaseResearch

A recent study emphasizes the importance of integrating patient-reported outcomes into cardiovascular care and research. This approach aims to enhance patient engagement and improve treatment outcomes.

embedding patient reported outcomes in cardiovascular care and research

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

Original title: “Embedding patient-reported outcomes in cardiovascular care and research.”

Previous262 / 486Next

Daily Briefings

Not rare diseases news,news about your rare disease.

Earlier

FDA Draft Guidance Could Streamline Rare Disease Gene Therapy Development | BioPharm International

EHMT1 Gene Therapy for Kleefstra Syndrome Enters Preclinical Collaboration | BioPharm International

Institute for Pediatric Rare Diseases at FSU, A.J. Anderson Foundation expand access to genetic screening and rare disease care - Florida State University News

Pelage Pharmaceuticals and Eli Lilly discuss innovative approaches to hair loss treatment, focusing on regenerative methods to reactivate dormant hair follicles. Early proof-of-concept findings could signal a shift in the long-stagnant hair loss treatment market.

A case report and literature review on primary large cell neuroendocrine carcinoma of the pulmonary artery has been published, contributing to the understanding of this rare cancer type. The findings may inform future research and clinical approaches.

A case report highlights autoimmune pulmonary alveolar proteinosis induced by brigatinib, contributing to the understanding of drug-related adverse effects. This literature review may inform clinicians about potential risks associated with brigatinib treatment.

A new study explores the pathogenesis of fibrosing mediastinitis, shedding light on common etiologies associated with this rare condition. Understanding these mechanisms may inform future therapeutic strategies.

IDefine and UT Southwestern Announce Research Collaboration to Advance Gene Therapy for Rare Disease Kleefstra Syndrome - BioSpace

A recent study reveals that 21 orofacial diseases share a common genetic architecture with systemic diseases, highlighting potential genetic links. This integrative genomic analysis could pave the way for new therapeutic strategies and insights into disease mechanisms.

A recent study utilizes machine learning to identify rare genetic variants that contribute to coronary artery disease risk in the Japanese population. This research enhances predictive capabilities, potentially guiding future genetic screening and intervention strategies.

AAV gene therapy for homozygous familial hypercholesterolemia: a phase 1 trial.

A new study expands the understanding of post-streptococcal neutrophilic pustular dermatosis, contributing to the histopathological framework of reactive neutrophilic dermatoses. This research may enhance diagnostic accuracy and treatment approaches for affected patients.

A case report details triple-vessel coronary involvement and a giant aneurysm in a 6-year-old boy with Kawasaki disease. This study highlights the severe cardiovascular complications that can arise from Kawasaki disease, emphasizing the need for early detection and management.

A rare case report details the co-occurrence of moyamoya syndrome and Turner syndrome in a 4-year-old girl from Sudan. This case highlights the complexities of diagnosing and managing multiple rare conditions simultaneously.

A recent study focuses on optimizing formulations of Levocarnitine for pediatric patients with inherited metabolic disorders. This research highlights the importance of tailored treatments to improve therapeutic outcomes in this vulnerable population.

A comprehensive review highlights granulomatous diseases that can mimic sarcoidosis, providing insights into differential diagnosis. This research may aid clinicians in recognizing and managing these conditions more effectively.

A new study reports the first identification of the BRAF V600E mutation in paratesticular mesothelioma, potentially opening avenues for targeted therapies. This discovery highlights the genetic underpinnings of this rare cancer type.

A pictorial review highlights interlobular septal thickening, providing insights into its radiological features. This review may aid in the diagnosis and understanding of related pulmonary conditions.

A 20-year pharmacovigilance study evaluates the safety profile of palivizumab, revealing insights from the FDA Adverse Event Reporting System. This research contributes to understanding the long-term effects of the drug in treating respiratory syncytial virus.

A recent study emphasizes the importance of integrating patient-reported outcomes into cardiovascular care and research. This approach aims to enhance patient engagement and improve treatment outcomes.

Not rare diseases news,
news about your rare disease.