Sammy Basso, Advocate for Progeria Research, Is Dead at 28Sammy Basso was a progeria advocate who lived humorously and courageously, raising awareness and contributing to research until his death at age 28.
Argentinian couple moves to US to allow their toddler to join gene therapy trialParents refused to give up despite rare disease diagnosis, traveled for gene therapy trial, showing hope and perseverance.
Lifesaving gene therapy for kids is world's priciest drug at $4.25MGene therapy approved for rare disease dramatically improves quality of life for children.High price of the therapy raises concerns about affordability and access.
Argentinian couple moves to US to allow their toddler to join gene therapy trialParents refused to give up despite rare disease diagnosis, traveled for gene therapy trial, showing hope and perseverance.
Lifesaving gene therapy for kids is world's priciest drug at $4.25MGene therapy approved for rare disease dramatically improves quality of life for children.High price of the therapy raises concerns about affordability and access.
Allergic angina (Kounis Syndrome) in mast cell disease patientsMCAS is a rare disease with only a few cases in certain states like Colorado.Some doctors may not be familiar with uncommon conditions like mast cell activation disease.
When our young son died, we decided to build him a boatThe story highlights the impact of a rare disease on a child's life and the power of imagination and adventure in coping with challenges.