First EB-Specific Treatment Now Available in Ireland: A Transformative Step for Patients In a move described as a “transformative moment” for patients, the Health Service Executive (HSE) has made the …
Tag:
rare diseases
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Stoke Therapeutics’ Zorevunersen Shows Promise in Treating Dravet Syndrome For children with Dravet syndrome, a rare and severe genetic epilepsy, developmental delays often begin after an initial period of normal …
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Adam Feuerstein Launches Biotech Newsletter – STAT’s Biotech Scorecard Adam Feuerstein, a senior writer and biotech columnist for STAT, has launched a weekly email newsletter called Adam’s Biotech Scorecard. The …
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Rare Disease Diagnosis: A Focus on the Hospital da Criança de Brasília The path to diagnosis can be long and arduous for families facing rare diseases. For Jakeline Carvalho, mother …
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Disorganized Nanoparticles May Be Key to More Effective Drug Delivery Lipid nanoparticles (LNPs) – the technology that powered the success of mRNA COVID-19 vaccines – may be more effective when …
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As the grandfather of a girl with a rare disease, I’ve learned that for families like ours, hope often comes not as a cure, but as a clinical trial: carefully …
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A virus long thought to be harmless may actually play a role in Parkinson’s disease, a condition that affects more than one million Americans.Northwestern Medicine scientists discovered Human Pegivirus (HPgV) …
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Long-Read Sequencing: A Beacon of Hope for Rare Disease Diagnosis For families navigating the complexities of rare genetic diseases, a diagnosis can feel elusive, a treasure hidden deep within the …