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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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ethnic inequities in mathematical models for respiratory infectious disease a systematic review
Original title: “Ethnic inequities in mathematical models for respiratory infectious disease: A systematic review.”
genomic clinical and radiological characterization of spontaneous cervical artery dissections findings from a single center cohort
Original title: “Genomic, clinical, and radiological characterization of spontaneous cervical artery dissections: Findings from a single-center cohort.”
extensive parenchymal brain calcification in a patient with autosomal dominant polycystic kidney disease and stage 5 chronic kidney disease
Original title: “Extensive Parenchymal Brain Calcification in a Patient With Autosomal Dominant Polycystic Kidney Disease and Stage 5 Chronic Kidney Disease.”
lymphangiogenesis driven by vegf c reshapes tumor immune landscape and enables tumor eradication by viral immunotherapy in mouse melanoma models
Original title: “Lymphangiogenesis driven by VEGF-C reshapes tumor immune landscape and enables tumor eradication by viral immunotherapy in mouse melanoma models.”
generalized megaesophagus concurrent with toxocariasis in a cat
Original title: “Generalized Megaesophagus Concurrent With Toxocariasis in a Cat.”
hla profiles and susceptibility to alveolar echinococcosis evidence from a large cohort with population and household controls
Original title: “HLA profiles and susceptibility to Alveolar echinococcosis: evidence from a large cohort with population and household controls.”
Explore the challenges of identifying and reaching rare disease patient populations throughout clinical development and commercialization · See how genomic, phenotypic, claims and EHR data can uncover patients, disease characteristics and healthcare journeys · Understand how patient-level insights can support cohort identification, clinical trial enrollment and clinical development decisions · Learn how insights generated across the therapy ... Explore the challenges of identifying and reaching rare disease patient populations throughout clinical development and commercialization · See how genomic, phenotypic, claims and EHR data can uncover patients, disease characteristics and healthcare journeys · Understand how patient-level insights can support cohort identification, clinical trial enrollment and clinical development decisions · Learn how insights generated across the therapy lifecycle can guide future development programs and support continuous discovery Biotech 1623 Clinical Trials 2376 Drug Discovery & Development 1625 Life Science 4693 Patient Recruitment & Retention 417 Pharma 2927 · Jump to every webinar tagged with a keyword from this one. Clinical Data Clinical Development Clinical Research Drug Development Genomic Data Genomics Patient Population Patient Recruitment Rare Diseases/Orphan Drugs Real-World Data Therapeutic Areas Join this webinar to explore how patient population data can improve rare disease patient identification and trial enrollment. Colleen Caleshu is a Clinician-Scientist Leader and Genetic Counselor with more than 23 years of experience in genomic medicine and real-world data, including over a decade collaborating with rare disease biopharma organizations. As Senior Director of Translational Research at GeneDx, she leverages GeneDx Infinity, the world’s largest clinically interpreted genomic dataset, to lead research and real-world evidence strategies that support biopharma partners across evidence generation, study design and clinical trial-enabling patient cohort development.
Original title: “Use Patient Population Data to Improve Therapy Lifecycle Decisions - Xtalks”
In a first, a subcutaneously administered RNA interference medicine crossed into the brain and plummeted levels of a protein implicated in the disease.
Original title: “Experimental Arrowhead RNAi drug hints at Alzheimer’s benefit”
Revolution Medicines’ recently approved drug showed a 24% overall response rate and a high rate of side effects in patients whose cancer has a G12D mutation, reinforcing Mizuho’s view that a more specific agent such as Verastem Oncology’s VS-7375 “should come first for G12D patients.”
Original title: “Verastem reports ‘robust’ response rate in pancreatic cancer subgroup where Rasonque struggled”
Wall Street analysts believe Verastem’s drug to now be “competitive” in what’s quickly become one of the most active and crowded areas of oncology research.
Original title: “Verastem cancer drug shows early promise in small study”
gch1 p ser80asn confers risk for parkinson s disease in east asian populations
Original title: “GCH1 p.Ser80Asn Confers Risk for Parkinson's Disease in East Asian Populations.”
it takes two to tango the intersection of deubiquitinases and m 6 a modification in cancer
Original title: “It takes two to tango: the intersection of deubiquitinases and m(6)A modification in cancer.”
orbital abscess complicating allergic fungal rhinosinusitis due to streptococcus halichoeri a rare zoonotic sinonasal infection
Original title: “Orbital Abscess Complicating Allergic Fungal Rhinosinusitis Due to Streptococcus halichoeri: A Rare Zoonotic Sinonasal Infection.”
intractable hiccups as initial manifestation of achalasia a case report
Original title: “Intractable hiccups as initial manifestation of achalasia: a case report.”
international recommendations for child to adult care transition in rare neurological diseases a scoping review and delphi consensus study
Original title: “International recommendations for child to adult care transition in rare neurological diseases: A scoping review and Delphi consensus study.”
financial burden to patients and informal carers of myelodysplastic syndromes in england a modelled analysis stratified by transfusion dependence
Original title: “Financial burden to patients and informal carers of myelodysplastic syndromes in England: a modelled analysis stratified by transfusion dependence.”
stereotactic body radiation therapy for management of locally advanced periorbital tumors efficacy and toxicity profile
Original title: “Stereotactic Body Radiation Therapy for Management of Locally Advanced Periorbital Tumors: Efficacy and Toxicity Profile.”