El CNIC recibe una donación para investigar el síndrome de progeria de Hutchinson-Gilford

by Centro Nacional de Investigaciones Cardiovasculares - CNIC

Esta donación ayudará a desarrollar la investigación del laboratorio del Dr. Andrés sobre el síndrome de progeria de Hutchinson-Gilford (del inglés HGPS), una enfermedad genética ultra-rara, co...

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A new CAR-T therapy shows efficacy in preclinical models of hard-to-treat leukemias

by Fundació Clínic per a la Recerca Biomèdica

Researchers from Clínic-IDIBAPS, within the Clínic Barcelona Comprehensive Cancer Center, and the spin-off Gyala Therapeutics have coordinated a study demonstrating the efficacy of CAR-T GYA01 thera...

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Understanding Immune Mechanisms Opens New Therapeutic Pathways in Autoimmune Neuropathies

by Hospital de la Santa Creu i Sant Pau

It has been analyzed recent advances in the diagnosis and treatment of primary autoimmune neuropathies—a group of rare and debilitating disorders of the peripheral nervous system that includes Guill...

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New pathway to treat a rare and aggressive liver cancer: Blocking CNNM4 protein with RNA technology

by CIC bioGUNE

The findings suggest that inhibiting CNNM4, combined with GalNAc siRNA technology, could offer a targeted, safe, and effective treatment, opening new avenues for personalized therapies against this ag...

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Veritas Genetics acquires CRG spinoff qGenomics

by Centre for Genomic Regulation

Veritas Genetics, a world leader in medical genomics, has acquired qGenomics, a leading company in carrier screening and array analysis. The scientific and technological expertise of qGenomics will al...

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ARTHEx Biotech Upsizes Series B Financing Round to $87 millones

by ARTHEx Biotech

Proceeds to support further clinical development of lead program ATX-01 in Myotonic Dystrophy Type1, a unique therapy currently in Phase I-IIa, targeting miR23b

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ORYZON to sponsor first Phelan-McDermid syndrome (PMS) burden of illness study

by Oryzon Genomics

Oryzon Genomics is proud to announce its participation as a sponsor of the first-ever Phelan-McDermid syndrome (PMS) burden of illness study. The study is led by CureShank, a research advocacy organiz...

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First evidence of a ‘nearly universal’ pharmacological chaperone for rare disease

by Centre for Genomic Regulation

A study published today in Nature Structural & Molecular Biology is the first time researchers have shown evidence that a single drug, already licensed for medical use, can stabilise nearly all mutate...

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El desarrollo de una terapia génica ‘suicida’ abre nuevas opciones contra el sarcoma de Ewing, un cáncer infantil raro

by CIBERER - Centro de Investigación Biomédica en Red de Enfermedades Raras

Cuando las células de sarcoma de Ewing son infectadas por el adenovirus terapéutico, estas expresan el gen ‘suicida’ HSV-TK. Este gen es inocuo, pero cuando se combina con la prodroga ganciclovi...

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Generative AI more efficient than nature at designing genome-editing proteins

by Centre for Genomic Regulation

It has been designed and experimentally validated new synthetic proteins that can edit the human genome more efficiently than proteins provided by nature that will be of great use in improving the cur...

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La AEMPS amplía el procedimiento de evaluación acelerada para ensayos de fases tempranas

by Sermes CRO

La Agencia Española de Medicamentos y Productos Sanitarios (AEMPS) ha anunciado la ampliación de su procedimiento de evaluación acelerada para ensayos clínicos en fases tempranas, una medida que c...

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IMPaCT-GENóMICA y CIBERER colaboran en el primer 'hackathon' en España que diagnostica casos de pacientes pediátricos con enfermedades raras

by CIBERER - Centro de Investigación Biomédica en Red de Enfermedades Raras

Otros objetivos del hackaton han sido demostrar la utilidad de nuevas técnicas para diagnosticar a pacientes complejos, establecer nuevos circuitos y redes de colaboración, así como sensibilizar so...

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