Zongertinib would be the first orally administered, targeted therapy for previously treated patients with HER2 (ERBB2)-mutant advanced non-small cell lung cancer (NSCLC), if approved
Menkes disease is a rare X-linked recessive pediatric disease caused by gene mutations of the copper transporter ATP7A
Aromatic L-amino acid decarboxylase deficiency is a rare genetic disorder that affects the production of some neurotransmitters
New indication supported by the MANDARA trial which showed nearly 60% of patients achieved remission and 41% of patients fully stopped taking oral corticosteroids
ALS patients experience neuroinflammation and rapid neurodegeneration
The initiation of a second Phase 3 clinical trial for bomedemstat demonstrates company’s commitment to advancing research in myeloproliferative neoplasms (MPNs)
Utidelone can penetrate BBB due to its unique physicochemical characteristics and insusceptibility to P-glycoprotein-mediated efflux
ALPHA Phase III trial showed first-in-class, oral, Factor D inhibitor as add-on to Ultomiris or Soliris improved haemoglobin levels and reduced anaemia and fatigue
NM5072 is an anti-Properdin antibody that selectively blocks the alternative pathway while maintaining the integrity of the classical pathway required for fending off infections
Utidelone has the ability to cross BBB due to its unique physicochemical characteristic and insusceptibility to P-glycoprotein-mediated efflux
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