A comprehensive view of Genetic Disorders. This page highlights a small sample of our full coverage.
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Premier Medical Laboratory Services adds noninvasive prenatal test that screens for chromosomal abnormalities such as Down Syndrome, indicates sex of baby, from blood sample; test is risk-free, highly accurate, covered by insurance for some pregnancies

Pfizer starting Phase 3 study in US of injectable fordadistrogene movaparvovec mini-dystrophin gene therapy for ambulatory patients with Duchenne muscular dystrophy after FDA lifts clinical hold on drug; DMD has very limited treatment options

Takeda’s injectable Takhzyro lanadelumab-flyo now available in US in single-dose prefilled syringes to prevent attacks of hereditary angioedema in adult, pediatric patients 12 years of age and older; HAE affects 1 in 50,000 people worldwide

FDA approves Novartis’ 170,000 square-foot, multi-product gene therapy manufacturing plant in Durham, North Carolina; plant to make injectable Zolgensma onasemnogene abeparvovec to treat spinal muscular atrophy, a leading genetic cause of infant death

Takeda announces approval in Japan for TAKHZYRO subcutaneous injection for prevention of acute attacks of hereditary angioedema; condition is a rare genetic disorder that causes recurring swelling

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