Steven White

Oct 16, 2024 | onlinelibrary.wiley.com | Liyan Wang |Wei Zhang |Steven White |Hang Fan

Corresponding Author Wei Zhang Department of Innovation, Entrepreneurship and Strategy, School of Economics and Management, Tsinghua University, Beijing, P. R. China Correspondence Wei Zhang, Department of Innovation, Entrepreneurship and Strategy, School of Economics and Management, Tsinghua University, Beijing 100084, P. R. China. Email: [email protected] for more papers by this author

Jun 5, 2024 | aei.org | Brian Miller |Steven White |Lauren MacDonald

For some parents, a visit to a doctor is a day forever burned in their memory. Rare pediatric diseases, or those affecting less than 200,000 children, collectively affect 10 million children in the US with only five percent of rare diseases with any FDA-approved treatment. Our country’s collective conscience is at risk with the looming expiration of a key program supporting the search for cures. Spinal Muscular Atrophy Type 1 (SMA) is one such single gene rare disease.