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  • Tarrytown, NY – Along with resolving site inspection issues that cost Regeneron a high-dose Eylea launch in June, the company has scored an FDA nod for Veopoz as the first treatment for the ultra-rare inherited immune condition Chaple disease. The disease, which is also known as CD55-deficient protein-losing enteropathy, has a global patient...
  • Manchester, UK – Babies and toddlers with a rare and fatal genetic condition can now receive life-saving treatment on the NHS for the first time. This follows clinical research studies first carried out at the National Institute for Health and Care Research (NIHR) Manchester Clinical Research Facility (CRF) at Royal...
  • Chicago, Ill. – The lab of Yongchao C. Ma, PhD, at Stanley Manne Children’s Research Institute at Ann & Robert H. Lurie Children’s Hospital of Chicago uncovered a novel mechanism that leads to motor neuron degeneration in spinal muscular atrophy (SMA). This discovery offers a new target for treatment that...

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