Our article on the first human clinical trial of a BBB molecular Trojan horse fusion protein just reached 40+ citations. Fusion proteins are a promising approach for neuronopathic lysosomal storage diseases. https://t.co/fXH0HZuqp5
Neurocognitive and somatic stabilization in pediatric patients with severe Mucopolysaccharidosis Type I after 52 weeks of intravenous brain-penetrating insulin receptor antibody-iduronidase fusion protein (valanafusp alpha): an open label phase 1-2 trial.
Neurocognitive and somatic stabilization in pediatric patients with severe Mucopolysaccharidosis Type I https://t.co/pSsihqgMub #mucopolisacaridosis #MPS #lisosomal #eerr #mucopolysaccharide #lysosomal #raredesease
MPS is a devastating disease. Giugliani & colleagues report the results of a clinical trial with a re-engineered iduronidase & note levels of glycosaminoglycans in CSF were not altered by treatment as compared with normal pediatric CSF supplied by
Neurocognitive and somatic stabilization in pediatric patients with severe Mucopolysaccharidosis Type I after ... https://t.co/jawnYmXzVp
Neurocognitive and somatic stabilization in pediatric patients with severe Mucopolysaccharidosis Type I after 52 weeks of intravenous brain-penetrating insulin receptor antibody-iduronidase fusion protein (valanafusp alpha): an open label phase 1-2 trial h
RT @bbbpapers: Neurocognitive and somatic stabilization in pediatric patients with severe Mucopolysaccharidosis Type I after 52 weeks of in…
Neurocognitive and somatic stabilization in pediatric patients with severe Mucopolysaccharidosis Type I after 52 weeks of intravenous brain-penetrating insulin receptor antibody-iduronidase fusion protein (valanafusp alpha): an open label phase 1-2 trial.