STAT Plus: Oh, Canada! Vertex battles another country over access to cystic fibrosis treatments
A row has broken out between Vertex Pharmaceuticals (VRTX) and Canadian officials over access to…
Category: rare disease
Opinion: Should ‘broken’ genes be fixed? My daughter changed the way I think about that question
I’m a doctor. I strive to fix things that are broken. Hearts, mostly. But my…
STAT Plus: Pharma execs criticize a drug maker over pricing and claim orphan drug law is being misused
Several prominent biotech and pharmaceutical executives are openly criticizing a huge price hike for a…
As medical professionals, my husband and I know the Nigerian travel ban is cruel. Now the pain is personal
A few days ago my daughter was diagnosed with a rare disease. In our time…
A lottery like no other offers up a cutting-edge medicine — with lives on the line
The lottery that began this week was not about money, or about choosing a school,…
If biotech had a Mount Rushmore, whose heads would you chisel in the granite?
If someone sculpted a Mount Rushmore of biotech CEOs, who’d be on it? The question…
Chan Zuckerberg Initiative to fund 30 patient groups, aiming to build a model for tackling rare diseases
SAN FRANCISCO — When Facebook founder Mark Zuckerberg and his spouse Dr. Priscilla Chan in…
STAT Plus: What does the latest twist in the Sarepta saga mean for the future of the FDA? We discuss
Earlier this week, the Food and Drug Administration released documents that reveal a schism within…
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STAT Plus: Blueprint gains approval for its first drug, a treatment for a rare gastrointestinal cancer
Blueprint Medicines won approval Thursday of its first drug, a precision medicine that dramatically shrank…
STAT Plus: Regeneron drug reduces bone growth for patients with ultra-rare skeletal disease
Patients with the ultra-rare disease FOP grow bone where it doesn’t belong, creating a life-threatening…
STAT Plus: As families in Europe clamor for its pricey gene therapy, Novartis creates a lottery
Seeking to provide its pricey gene therapy to patients in countries where the drug is…
Waning treatment for immune deficiency is a warning for all ‘one-and-done’ therapies
As a new mother, she didn’t know to look for blue-tinged lips. She could just…
FDA changes course on Sarepta’s once-rejected muscular dystrophy drug
The Food and Drug Administration reversed its decision on a treatment for Duchenne muscular dystrophy…
Families are reeling after FDA rejects therapy for kids born without a thymus gland
When Charlie Luckesen turned 2 the Wednesday before Thanksgiving, his family celebrated with a golden…
A clinical trial for Huntington’s opens the door to hope for some patients. Only so many can get in
PERRY, N.Y. — Reminders of Huntington’s disease populate Tammy Stewart’s apartment. They are also glimpses…