Astellas' Breakthrough Treatment Revives Hope for Rare Disease Patients and Families
Astellas Pharma's experimental treatment for a rare, devastating disease has reignited hope among patients and advocates. The breakthrough comes amid...
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Five Years After Deadly Disaster, Rare Disease Community Secures New Treatment Hope
Joshua Jacob Gonzalez’s parents suctioned his saliva every five minutes to prevent choking. After a groundbreaking treatment, they noticed a life-chan...
Intellia’s CRISPR Gene Therapy for Rare Disease Advances in Phase 3 Trial, Sun Pharma to Acquire Organon for $11.75B
Intellia Therapeutics reported that a single dose of its CRISPR-based gene editing therapy, lonvo-z, dramatically reduced swelling attacks in a Phase...