A Loophole Brings Cystic Fibrosis Patients a ‘Miracle Drug’ in Generic Form
A generic version of a breakthrough cystic fibrosis drug, manufactured [...]
2025 Lasker Awards Given to Cell Biology and Cystic Fibrosis Pioneers
Many winners of the annual Lasker Awards have gone on [...]
People With Severe Diabetes Are Cured in Small Trial of New Drug
Most in a small group of patients receiving a stem [...]
Boston Faces Identity Crisis Amid Trump’s Cuts to University and Research Funding
Colleges and teaching hospitals are the cornerstones of the city’s [...]
FDA Approves Journavx Drug to Treat Pain Without Addiction Risk
The drug, Journavx by Vertex Pharmaceuticals, blocks pain signals to [...]
F.D.A. Approves 2 Sickle Cell Treatments, One Using CRISPR Gene Editing
People with the genetic disease have new opportunities to eliminate [...]
New Sickle Cell Therapies Will Be Out of Reach Where They Are Needed Most
There is no clear path for African patients to get [...]
Sickle-Cell Treatment Created With Gene Editing Wins U.K. Approval
The first treatment that relies on CRISPR is expected to [...]
Panel Tells F.D.A. That CRISPR Sickle Cell Cure Is Safe Enough for Patients
The decision by an advisory committee may lead to Food [...]
F.D.A. Experts Will Vote on Safety of a Cure for Sickle Cell Disease
The treatment from the company Vertex would be the first [...]