Researchers identify genetic changes responsible for rare childhood immune disorders
Affecting the immune system, various forms of SCID are estimated to affect one in 75,000 to 100,000 newborns
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Tag: rare genetic disorders
MHRA approves Marinus Pharmaceuticals’ Ztalmy for CDKL5 deficiency disorder
The rare epileptic seizure disorder currently affects around one in 42,000 people in the UK
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- Acesion Pharma launches phase 2 trial of AP31969 in atrial fibrillation
- Johnson & Johnson seeks EMA approval for teclistamab–daratumumab combination
- HUTCHMED begins phase 3 stage of pancreatic cancer trial
- Zenas reports strong phase 3 results for obexelimab in IgG4-related disease
- MRM Health collaborates with Oncode Institute and Netherlands Cancer Institute
