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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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Daily Briefings

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Today

15 stories

Research

Ultragenyx prices Sanfilippo gene therapy at $4M; Bristol Myers shelves Orum drug

BioPharma Dive·about 8 hours ago·Read at source →

Research

Ultragenyx wins FDA greenlight for first Sanfilippo therapy, priced at nearly $4M

BioSpace·about 10 hours ago·Read at source →

lymphedema-distichiasis syndromeResearch

A recent study published in PubMed details the occurrence of corneal sequestration and bilateral distichiasis in a binturong, contributing to the understanding of these conditions in non-traditional animal models. This research may provide insights into similar ocular conditions in other species.

corneal sequestration and bilateral distichiasis in a binturong arctictis binturong

PubMed Rare Disease·about 10 hours ago·Read at source →

Original title: “Corneal Sequestration and Bilateral Distichiasis in a Binturong (Arctictis binturong).”

Research

A case study highlights the diagnostic challenges of primary pulmonary low-grade fibromyxoid sarcoma in a 70-year-old woman, emphasizing the complexities in thoracic spindle cell pathology. This research contributes to the understanding of rare sarcomas and their clinical presentation.

primary pulmonary low grade fibromyxoid sarcoma in a 70 year old woman a diagnostic challenge in thoracic spindle cell pathology

PubMed Rare Disease·about 22 hours ago·Read at source →

Original title: “Primary Pulmonary Low-Grade Fibromyxoid Sarcoma in a 70-Year-Old Woman: A Diagnostic Challenge in Thoracic Spindle Cell Pathology.”

sarcoma sarcoma G1Research

A new study highlights the role of multimodality imaging in managing primary cardiac undifferentiated pleomorphic sarcoma, which can present as obstructive shock. This research may enhance diagnostic and treatment strategies for this rare cancer.

primary cardiac undifferentiated pleomorphic sarcoma presenting as obstructive shock multimodality imaging guides management

PubMed Rare Disease·about 22 hours ago·Read at source →

Original title: “Primary Cardiac Undifferentiated Pleomorphic Sarcoma Presenting as Obstructive Shock: Multimodality Imaging Guides Management.”

penile cancer penile urethral cancerResearch

A phase II trial, MH-Penile-002, investigates a neoadjuvant treatment combining an anti-EGFR antibody-drug conjugate with a PD-1 inhibitor for penile cancer. This study focuses on patients facing challenges with penile preservation or regional lymph node metastasis.

mh penile 002 a phase ii prospective single arm trial of neoadjuvant anti egfr antibody drug conjugate plus pd 1 inhibitor in penile cancer with challenging penile preservation or regional lymph node metastasis

PubMed Rare Disease·about 22 hours ago·Read at source →

Original title: “MH-Penile-002: a phase II, prospective, single-arm trial of neoadjuvant anti-EGFR antibody-drug conjugate plus PD-1 inhibitor in penile cancer with challenging penile preservation or regional lymph node metastasis.”

paracoccidioidomycosisResearch

A case study highlights ileocecal paracoccidioidomycosis in a pediatric patient, presenting as a pseudotumoral lesion. This case underscores the diagnostic challenges associated with this rare fungal infection.

ileocecal paracoccidioidomycosis presenting as a pseudotumoral lesion in a pediatric patient a histopathological diagnostic challenge

PubMed Rare Disease·about 22 hours ago·Read at source →

Original title: “Ileocecal Paracoccidioidomycosis Presenting as a Pseudotumoral Lesion in a Pediatric Patient: A Histopathological Diagnostic Challenge.”

pulmonary hypertension pulmonary hypertension, primary, 6+1 moreResearch

A recent EUSTAR cohort study evaluates the effectiveness of oral anticoagulants in treating precapillary pulmonary hypertension linked to systemic sclerosis. The findings contribute to understanding treatment options for this rare condition.

effectiveness of oral anticoagulants in precapillary pulmonary hypertension associated with systemic sclerosis a eustar cohort study

PubMed Rare Disease·about 22 hours ago·Read at source →

Original title: “Effectiveness of oral anticoagulants in precapillary pulmonary hypertension associated with systemic sclerosis: a EUSTAR cohort study.”

herpes simplex virus gingivostomatitis herpes simplex virus keratitis+1 moreResearch

A case report highlights a multifocal mucocutaneous herpes simplex virus type 1 infection occurring in a young adult following seasonal influenza vaccination. This observation raises questions about potential vaccine-related immune responses in individuals without known immunocompromising conditions.

multifocal mucocutaneous herpes simplex virus type 1 infection temporally associated with seasonal influenza vaccination in a young adult without a known immunocompromising condition a case report

PubMed Rare Disease·about 22 hours ago·Read at source →

Original title: “Multifocal mucocutaneous herpes simplex virus type 1 infection temporally associated with seasonal influenza vaccination in a young adult without a known immunocompromising condition: A case report.”

Research

A multicenter study evaluates the electrical characteristics and short-term outcomes of the Aveir VR leadless pacemaker. The findings contribute to understanding the device's performance in patients requiring cardiac pacing.

multicenter study of the electrical characteristics and short term outcomes of the aveir vr leadless pacemaker

PubMed Rare Disease·about 22 hours ago·Read at source →

Original title: “Multicenter Study of the Electrical Characteristics and Short-Term Outcomes of the Aveir VR Leadless Pacemaker.”

ovarian cancerResearch

A rare case study highlights abdominal actinomycosis presenting symptoms similar to ovarian cancer, with potential hepatic and abdominal wall involvement. This discovery may enhance diagnostic accuracy for similar cases.

rare case of abdominal actinomycosis mimicking ovarian cancer with presumed hepatic involvement and abdominal wall involvement

PubMed Rare Disease·about 22 hours ago·Read at source →

Original title: “A rare case of abdominal actinomycosis mimicking ovarian cancer with presumed hepatic involvement and abdominal wall involvement.”

adult-onset Still diseaseResearch

A recent study highlights the effectiveness of skin biopsy as a rapid and specific diagnostic test for adult-onset Still's disease. This method offers a widely available option for clinicians to improve diagnosis in patients presenting with this rare condition.

skin biopsy in adult onset still s disease a specific rapid and widely available diagnostic test

PubMed Rare Disease·about 22 hours ago·Read at source →

Original title: “Skin biopsy in adult-onset Still's disease: A specific, rapid, and widely available diagnostic test.”

Research

A narrative review examines neonatal and long-term outcomes for infants born to mothers who underwent solid organ transplantation, utilizing data from a Polish cohort. The findings contribute to understanding the implications of maternal organ transplants on infant health.

neonatal and long term outcomes in infants born to mothers after solid organ transplantation a narrative review with polish cohort data

PubMed Rare Disease·about 22 hours ago·Read at source →

Original title: “Neonatal and long-term outcomes in infants born to mothers after solid organ transplantation: a narrative review with Polish cohort data.”

Moyamoya disease moyamoya disease 7+1 moreResearch

A recent meta-regression study explores the prediction of cerebral hyperperfusion syndrome following STA-MCA bypass in patients with moyamoya disease using CT perfusion techniques. This research could enhance surgical outcomes and patient management strategies.

predicting cerebral hyperperfusion syndrome after sta mca bypass in moyamoya disease with ct perfusion through meta regression

PubMed Rare Disease·about 22 hours ago·Read at source →

Original title: “Predicting cerebral hyperperfusion syndrome after STA-MCA bypass in Moyamoya disease with CT perfusion through meta-regression.”

Research

A recent study highlights black pleural effusion as a rare manifestation of serotype 3 pneumococcal empyema. This finding may prompt further investigation into atypical presentations of this condition.

black pleural effusion as a rare presentation of serotype 3 pneumococcal empyema

PubMed Rare Disease·about 22 hours ago·Read at source →

Original title: “Black Pleural Effusion as a Rare Presentation of Serotype 3 Pneumococcal Empyema.”

Yesterday

5 stories

wet macular degeneration age related macular degeneration 7Research

Experts discuss the challenges of treating wet age-related macular degeneration (AMD) with current anti-VEGF therapies and emphasize the need for more durable treatment options. The conversation highlights the potential for advancements that could significantly improve patient outcomes.

In this episode of Denatured, you’ll hear Philip Jacob Rosenfeld, professor of Ophthalmology at the University of Miami Health System and Bascom Palmer Eye Institute, and Wesley Jackson, chief science officer at Valitor. We talk about the eye condition call wet age-related macular degeneration (AMD), the limits of anti‑VEGF therapy and the push for true durability to change a patient’s life.

BioSpace·1 day ago·Read at source →

Original title: “When vision fades: Rethinking wet AMD and the durability gap”

Research

EpilepsyGTx and Viralgen partner to scale AAV manufacturing for EPY201

Pharma Times·1 day ago·Read at source →

lysosomal storage disease atherosclerotic cardiovascular diseaseResearch

Mexico Cell and Gene Therapy Market Set for Expansion Through 2034 as Precision Medicine Advances, Clinical Investment Rises, and Innovative Delivery Models Gain Momentum

Gene & Cell Therapy·1 day ago·Read at source →

penile cancer penile urethral cancerResearch

The PERICLES trial identifies pre-existing immunity and high-risk HPV status as key factors influencing immunotherapy response in advanced penile cancer. These findings could guide future treatment strategies and patient selection.

pre existing immunity and hrhpv status determine immunotherapy response in advanced penile cancer biomarkers and overall survival in the pericles trial

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

Original title: “Pre-existing immunity and hrHPV status determine immunotherapy response in advanced penile cancer: biomarkers and overall survival in the PERICLES trial.”

pantothenate kinase-associated neurodegeneration atypical pantothenate kinase-associated neurodegeneration+1 moreResearch

A new study demonstrates that a selective PanK3 activator successfully restores coenzyme A levels in cellular and mouse models of pantothenate kinase-associated neurodegeneration. This research could pave the way for novel therapeutic strategies targeting this rare neurodegenerative condition.

selective pank3 activator restores coenzyme a in cellular and mouse models of pantothenate kinase associated neurodegeneration

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

Original title: “A Selective PanK3 Activator Restores Coenzyme A in Cellular and Mouse Models of Pantothenate Kinase-Associated Neurodegeneration.”

1 / 388Next

Daily Briefings

Not rare diseases news,news about your rare disease.

Today

Ultragenyx prices Sanfilippo gene therapy at $4M; Bristol Myers shelves Orum drug

Ultragenyx wins FDA greenlight for first Sanfilippo therapy, priced at nearly $4M

A recent study published in PubMed details the occurrence of corneal sequestration and bilateral distichiasis in a binturong, contributing to the understanding of these conditions in non-traditional animal models. This research may provide insights into similar ocular conditions in other species.

A case study highlights the diagnostic challenges of primary pulmonary low-grade fibromyxoid sarcoma in a 70-year-old woman, emphasizing the complexities in thoracic spindle cell pathology. This research contributes to the understanding of rare sarcomas and their clinical presentation.

A new study highlights the role of multimodality imaging in managing primary cardiac undifferentiated pleomorphic sarcoma, which can present as obstructive shock. This research may enhance diagnostic and treatment strategies for this rare cancer.

A phase II trial, MH-Penile-002, investigates a neoadjuvant treatment combining an anti-EGFR antibody-drug conjugate with a PD-1 inhibitor for penile cancer. This study focuses on patients facing challenges with penile preservation or regional lymph node metastasis.

A case study highlights ileocecal paracoccidioidomycosis in a pediatric patient, presenting as a pseudotumoral lesion. This case underscores the diagnostic challenges associated with this rare fungal infection.

A recent EUSTAR cohort study evaluates the effectiveness of oral anticoagulants in treating precapillary pulmonary hypertension linked to systemic sclerosis. The findings contribute to understanding treatment options for this rare condition.

A multicenter study evaluates the electrical characteristics and short-term outcomes of the Aveir VR leadless pacemaker. The findings contribute to understanding the device's performance in patients requiring cardiac pacing.

A rare case study highlights abdominal actinomycosis presenting symptoms similar to ovarian cancer, with potential hepatic and abdominal wall involvement. This discovery may enhance diagnostic accuracy for similar cases.

A recent study highlights the effectiveness of skin biopsy as a rapid and specific diagnostic test for adult-onset Still's disease. This method offers a widely available option for clinicians to improve diagnosis in patients presenting with this rare condition.

A narrative review examines neonatal and long-term outcomes for infants born to mothers who underwent solid organ transplantation, utilizing data from a Polish cohort. The findings contribute to understanding the implications of maternal organ transplants on infant health.

A recent meta-regression study explores the prediction of cerebral hyperperfusion syndrome following STA-MCA bypass in patients with moyamoya disease using CT perfusion techniques. This research could enhance surgical outcomes and patient management strategies.

A recent study highlights black pleural effusion as a rare manifestation of serotype 3 pneumococcal empyema. This finding may prompt further investigation into atypical presentations of this condition.

Yesterday

Experts discuss the challenges of treating wet age-related macular degeneration (AMD) with current anti-VEGF therapies and emphasize the need for more durable treatment options. The conversation highlights the potential for advancements that could significantly improve patient outcomes.

EpilepsyGTx and Viralgen partner to scale AAV manufacturing for EPY201

Mexico Cell and Gene Therapy Market Set for Expansion Through 2034 as Precision Medicine Advances, Clinical Investment Rises, and Innovative Delivery Models Gain Momentum

The PERICLES trial identifies pre-existing immunity and high-risk HPV status as key factors influencing immunotherapy response in advanced penile cancer. These findings could guide future treatment strategies and patient selection.

A new study demonstrates that a selective PanK3 activator successfully restores coenzyme A levels in cellular and mouse models of pantothenate kinase-associated neurodegeneration. This research could pave the way for novel therapeutic strategies targeting this rare neurodegenerative condition.

Not rare diseases news,
news about your rare disease.