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News about "genetic disorder "
FDA Approves Regeneron's Pasatru to Reduce New Bone Formation in FOP
Regeneron’s Pasatru has received FDA approval for adults with FOP, with Phase 3 OPTIMA data showing up to a 94 percent reduction in new heterotopic ossification lesions compared with placebo.
Genetic Disorder | 24/08/2026 | By News Bureau | 606
Sanofi's Nexviazyme Meets Primary Goal in Phase 3 Study for Infantile-Onset Pompe Disease
Sanofi's Phase 3 Baby-COMET study showed Nexviazyme improved ventilator-free survival in infants with infantile-onset Pompe disease, supporting plans for a US regulatory filing to expand the therapy's approved indication.
Genetic Disorder | 01/07/2026 | By News Bureau | 158
Pint Pharma Announces Approval of ORLADEYO in Peru
Pint Pharma has announced that the General Directorate of Medicines Supplies and Drugs (DIGEMID) in Peru has announced the approval of ORLADEYO (berotralstat) for prophylaxis of hereditary angioedema (HAE) attacks in adults and children 12 years and older.
Genetic Disorder | 10/07/2024 | By Aishwarya | 758
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