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  • More than a Million Children Provide Familial Care in the US
  • Trial Testing EDV Nanocells in Pancreatic, Other EGFR-positive Advanced Solid Cancers Now Dosing Patients
  • How Zolgensma Works and Trials Underway in Range of SMA Patients
  • Romanian Mom Leads Nationwide Effort to Get Spinraza Funded for Daughter and All SMA Patients
  • Agency Unveils RaDaR to Help Patient Groups Develop Rare Disease Registries
  • 31 Days of CF: William’s Story
  • After Considering Patients’ Well-being, Rubraca Continues to Benefit Women with Recurrent Ovarian Cancer, Analysis Shows
  • FDA Approves Zolgensma, 1st Gene Therapy to Treat SMA in Children Up to Age 2
  • Patients with Bipolar Disorder at Higher Risk of Developing Parkinson’s, Study Suggests
  • Post-transplant Holidays and Milestones Are Causes to Celebrate and to Grieve