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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

Arrowhead Pharmaceuticals presents new findings on Redemplo for severe hypertriglyceridemia, highlighting safety and dosing as potential differentiators against Ionis Pharmaceuticals. Analysts remain divided on the implications for market competition.

Safety and dosing are likely to be “key differentiators” for Arrowhead Pharmaceuticals as it looks to challenge Ionis Pharmaceuticals’ lead in the severe hypertriglyceridemia market.

BioSpace·2 months ago·Read at source →

Original title: “Arrowhead's new Redemplo findings in severe hypertriglyceridemia have analysts split”

Research

Carterra's HT-SPR platforms facilitate a study validating 1,320 binders across 15 targets, showcasing advancements in AI-designed protein research. This collaboration with Anthropic, Twist, and Adaptyv highlights the growing intersection of AI and biotechnology.

Anthropic, twist and Adaptyv validate 1,320 binders across 15 targets

Pharma Times·2 months ago·Read at source →

Original title: “Carterra supports AI‑designed protein study with HT-SPR platforms”

Research

The phase 3 Vivacity-MG3 study demonstrates the efficacy of nipocalimab in treating moderate-to-severe ocular symptoms in patients with generalized myasthenia gravis. This research highlights a potential new therapeutic option for this challenging condition.

efficacy of nipocalimab in patients with moderate to severe ocular symptoms of generalized myasthenia gravis in the phase 3 vivacity mg3 study

PubMed Rare Disease·2 months ago·Read at source →

Original title: “Efficacy of nipocalimab in patients with moderate-to-severe ocular symptoms of generalized myasthenia gravis in the phase 3 Vivacity-MG3 study.”

multiple system atrophy multiple system atrophy, parkinsonian typeResearch

Recent research highlights the potential of plasma neurofilament light as a biomarker for early differentiation between multiple system atrophy and Parkinson's disease. This discovery could enhance diagnostic accuracy and improve patient management strategies.

plasma neurofilament light for early differentiation of multiple system atrophy from parkinson disease

PubMed Rare Disease·2 months ago·Read at source →

Original title: “Plasma neurofilament light for early differentiation of multiple system atrophy from Parkinson disease.”

mucosal melanomaResearch

Recent genomic profiling of mucosal melanoma has identified novel fusion transcripts and highlighted dysregulation in key pathways, including cell cycle, MAPK, and PI3K. These findings could inform future therapeutic strategies for this rare cancer.

comprehensive genomic profiling of mucosal melanoma reveals novel fusion transcripts and dysregulation of cell cycle mapk and pi3k pathways

PubMed Rare Disease·2 months ago·Read at source →

Original title: “Comprehensive genomic profiling of mucosal melanoma reveals novel fusion transcripts and dysregulation of cell-cycle, MAPK, and PI3K pathways.”

lymphangioleiomyomatosis lung lymphangioleiomyomatosis+1 moreResearch

A case report highlights the multidisciplinary management of lymphangioleiomyomatosis in a pregnant patient experiencing severe bilateral pneumothorax. This study underscores the complexities of treating rare diseases during pregnancy.

multidisciplinary management of lymphangioleiomyomatosis presenting with severe bilateral pneumothorax during pregnancy a case report

PubMed Rare Disease·2 months ago·Read at source →

Original title: “Multidisciplinary Management of Lymphangioleiomyomatosis Presenting With Severe Bilateral Pneumothorax During Pregnancy: A Case Report.”

Research

A recent study published in PubMed examines the clinical outcomes of low-grade appendiceal mucinous neoplasms at a single institution. The findings contribute to the understanding of this rare condition, although specific outcomes and data were not detailed in the excerpt.

clinical outcomes of low grade appendiceal mucinous neoplasms in a single institution

PubMed Rare Disease·2 months ago·Read at source →

Original title: “Clinical Outcomes of Low-Grade Appendiceal Mucinous Neoplasms in a Single Institution.”

Research

A recent study published in PubMed examines the accuracy and beliefs of investment professionals regarding rare disease benchmarks. The findings highlight the need for improved understanding and data in this niche sector.

rare disease benchmarks investment professionals accuracy and beliefs

PubMed Rare Disease·2 months ago·Read at source →

Original title: “Rare disease benchmarks: investment professionals' accuracy and beliefs.”

hypertriglyceridemia hypertriglyceridemia 2+1 moreResearch

A rare case study highlights the relationship between hypertriglyceridemia, chronic kidney disease, and proteinuria in a patient with apolipoprotein C-II amyloidosis. This research contributes to the understanding of the disease's impact on kidney function.

hypertriglyceridemia chronic kidney disease and proteinuria a rare case of apolipoprotein c ii amyloidosis

PubMed Rare Disease·2 months ago·Read at source →

Original title: “[Hypertriglyceridemia, Chronic Kidney Disease, and Proteinuria: A Rare Case of Apolipoprotein C-II Amyloidosis].”

anal canal cancer anal canal carcinoma+1 moreResearch

Recent research explores therapeutic approaches for metastatic or unresectable recurrent anal canal cancer, highlighting emerging perspectives in treatment strategies. This study contributes to the understanding of this rare cancer's management.

metastatic or unresectable recurrent anal canal cancer therapeutic approaches and emerging perspectives

PubMed Rare Disease·2 months ago·Read at source →

Original title: “[Metastatic or unresectable recurrent anal canal cancer: Therapeutic approaches and emerging perspectives].”

melioidosisResearch

A recent study published in PubMed investigates the seroprevalence of Burkholderia pseudomallei among international travelers, highlighting potential exposure risks. This research contributes to understanding the epidemiology of melioidosis, a disease caused by this pathogen.

seroprevalence of burkholderia pseudomallei among international travellers

PubMed Rare Disease·2 months ago·Read at source →

Original title: “Seroprevalence of Burkholderia pseudomallei Among International Travellers.”

lung cancer lung cancer susceptibility 4+1 moreResearch

A comprehensive review highlights the evolution of lung cancer screening over the past sixty years, transitioning from sputum cytology to low-dose CT. The article discusses the implications of emerging precision medicine approaches in improving early detection and outcomes.

sixty years of lung cancer screening from sputum cytology to low dose ct and the emerging precision era

PubMed Rare Disease·2 months ago·Read at source →

Original title: “Sixty Years of Lung Cancer Screening: From Sputum Cytology to Low-Dose CT and the Emerging Precision Era.”

Erdheim-Chester diseaseResearch

A recent study highlights a rare presentation of primary breast involvement in Erdheim-Chester disease, expanding the understanding of this condition. This discovery may influence future diagnostic and treatment approaches for affected patients.

primary breast involvement in erdheim chester disease a rare presentation

PubMed Rare Disease·2 months ago·Read at source →

Original title: “Primary Breast Involvement in Erdheim-Chester Disease: A Rare Presentation.”

Research

A new study explores the use of intrapulmonary percussive ventilation for managing lung damage in patients with Swyer-James-MacLeod syndrome. This research could provide insights into innovative treatment approaches for this rare condition.

intrapulmonary percussive ventilation for the management of destroyed lung in swyer james macleod syndrome

PubMed Rare Disease·2 months ago·Read at source →

Original title: “Intrapulmonary percussive ventilation for the management of destroyed lung in Swyer-James-MacLeod syndrome.”

Research

A recent case report highlights digital acro-osteolysis as a revealing symptom of Hajdu-Cheney syndrome in an elderly patient. This discovery adds to the understanding of the clinical presentation of this rare genetic disorder.

digital acro osteolysis revealing hajdu cheney syndrome in an elderly patient a case report

PubMed Rare Disease·2 months ago·Read at source →

Original title: “Digital Acro-osteolysis Revealing Hajdu-Cheney Syndrome in an Elderly Patient: A Case Report.”

Research

A new framework called the therapeutic mechanism-trajectories principle (TMTP) has been proposed to enhance informational architecture in chronic disease self-management. This framework aims to improve patient engagement and outcomes in managing chronic conditions.

therapeutic mechanism trajectories principle tmtp a framework for informational architecture in chronic disease self management

PubMed Rare Disease·2 months ago·Read at source →

Original title: “The therapeutic mechanism-trajectories principle (TMTP): A framework for informational architecture in chronic disease self-management.”

IgG4-related thyroid diseaseResearch

A recent case study highlights multinervous cranial involvement in an adult patient with IgG4-related disease, affecting facial, infraorbital, auriculotemporal, and optic nerves. This research adds to the understanding of the disease's neurological manifestations.

igg4 related disease involving facial infraorbital auriculotemporal and optic nerves multinervous cranial involvement in an adult patient

PubMed Rare Disease·2 months ago·Read at source →

Original title: “IgG4-related disease involving facial, infraorbital, auriculotemporal and optic nerves: multinervous cranial involvement in an adult patient.”

dermatitis epilepsyResearch

A rare case study published in PubMed highlights dermatitis artefacta in an epileptic female, illustrating the complex interplay between mental health and skin conditions. This case contributes to the understanding of psychosomatic disorders in rare disease contexts.

when mind hurts the skin a rare case of dermatitis artefacta in an epileptic female

PubMed Rare Disease·2 months ago·Read at source →

Original title: “When Mind Hurts the Skin: A Rare Case of Dermatitis Artefacta in an Epileptic Female.”

Myhre syndrome ruvalcaba churesigaew myhre syndromeResearch

Recent research highlights advancements in clinical care and therapeutic strategies for Myhre syndrome. The findings aim to improve patient outcomes and guide future studies in this rare genetic disorder.

myhre syndrome new horizons in clinical care research and the pursuit of therapy

PubMed Rare Disease·2 months ago·Read at source →

Original title: “Myhre Syndrome: New Horizons in Clinical Care, Research and the Pursuit of Therapy.”

neuromuscular diseaseResearch

A new publication explores the potential of artificial intelligence to address challenges in neuromuscular diseases, a field often limited by data scarcity. The study highlights opportunities for improving diagnosis and treatment through advanced data analysis techniques.

artificial intelligence in neuromuscular diseases opportunities for a data scarce field

PubMed Rare Disease·2 months ago·Read at source →

Original title: “Artificial Intelligence in Neuromuscular Diseases: Opportunities for a Data-Scarce Field.”

Previous128 / 482Next

Daily Briefings

Not rare diseases news,news about your rare disease.

Earlier

Arrowhead Pharmaceuticals presents new findings on Redemplo for severe hypertriglyceridemia, highlighting safety and dosing as potential differentiators against Ionis Pharmaceuticals. Analysts remain divided on the implications for market competition.

Carterra's HT-SPR platforms facilitate a study validating 1,320 binders across 15 targets, showcasing advancements in AI-designed protein research. This collaboration with Anthropic, Twist, and Adaptyv highlights the growing intersection of AI and biotechnology.

The phase 3 Vivacity-MG3 study demonstrates the efficacy of nipocalimab in treating moderate-to-severe ocular symptoms in patients with generalized myasthenia gravis. This research highlights a potential new therapeutic option for this challenging condition.

Recent research highlights the potential of plasma neurofilament light as a biomarker for early differentiation between multiple system atrophy and Parkinson's disease. This discovery could enhance diagnostic accuracy and improve patient management strategies.

Recent genomic profiling of mucosal melanoma has identified novel fusion transcripts and highlighted dysregulation in key pathways, including cell cycle, MAPK, and PI3K. These findings could inform future therapeutic strategies for this rare cancer.

A case report highlights the multidisciplinary management of lymphangioleiomyomatosis in a pregnant patient experiencing severe bilateral pneumothorax. This study underscores the complexities of treating rare diseases during pregnancy.

A recent study published in PubMed examines the clinical outcomes of low-grade appendiceal mucinous neoplasms at a single institution. The findings contribute to the understanding of this rare condition, although specific outcomes and data were not detailed in the excerpt.

A recent study published in PubMed examines the accuracy and beliefs of investment professionals regarding rare disease benchmarks. The findings highlight the need for improved understanding and data in this niche sector.

A rare case study highlights the relationship between hypertriglyceridemia, chronic kidney disease, and proteinuria in a patient with apolipoprotein C-II amyloidosis. This research contributes to the understanding of the disease's impact on kidney function.

Recent research explores therapeutic approaches for metastatic or unresectable recurrent anal canal cancer, highlighting emerging perspectives in treatment strategies. This study contributes to the understanding of this rare cancer's management.

A recent study published in PubMed investigates the seroprevalence of Burkholderia pseudomallei among international travelers, highlighting potential exposure risks. This research contributes to understanding the epidemiology of melioidosis, a disease caused by this pathogen.

A comprehensive review highlights the evolution of lung cancer screening over the past sixty years, transitioning from sputum cytology to low-dose CT. The article discusses the implications of emerging precision medicine approaches in improving early detection and outcomes.

A recent study highlights a rare presentation of primary breast involvement in Erdheim-Chester disease, expanding the understanding of this condition. This discovery may influence future diagnostic and treatment approaches for affected patients.

A new study explores the use of intrapulmonary percussive ventilation for managing lung damage in patients with Swyer-James-MacLeod syndrome. This research could provide insights into innovative treatment approaches for this rare condition.

A recent case report highlights digital acro-osteolysis as a revealing symptom of Hajdu-Cheney syndrome in an elderly patient. This discovery adds to the understanding of the clinical presentation of this rare genetic disorder.

A new framework called the therapeutic mechanism-trajectories principle (TMTP) has been proposed to enhance informational architecture in chronic disease self-management. This framework aims to improve patient engagement and outcomes in managing chronic conditions.

A recent case study highlights multinervous cranial involvement in an adult patient with IgG4-related disease, affecting facial, infraorbital, auriculotemporal, and optic nerves. This research adds to the understanding of the disease's neurological manifestations.

A rare case study published in PubMed highlights dermatitis artefacta in an epileptic female, illustrating the complex interplay between mental health and skin conditions. This case contributes to the understanding of psychosomatic disorders in rare disease contexts.

Recent research highlights advancements in clinical care and therapeutic strategies for Myhre syndrome. The findings aim to improve patient outcomes and guide future studies in this rare genetic disorder.

A new publication explores the potential of artificial intelligence to address challenges in neuromuscular diseases, a field often limited by data scarcity. The study highlights opportunities for improving diagnosis and treatment through advanced data analysis techniques.

Not rare diseases news,
news about your rare disease.