rare diseases

Adam Feuerstein, Allison DeAngelis, Damian Garde, and Matthew Herper

STAT Plus: This is the TLDR of JPM Day 1

Biotech Damian Garde

STAT Plus: FDA approves AstraZeneca, Ionis treatment for rare nerve disease

Morning Rounds Elizabeth Cooney

Morning Rounds: Why health care costs are nearly a fifth of the GDP

The Readout Damian Garde

STAT Plus: BioMarin appeases an activist

The Readout Meghana Keshavan

STAT Plus: The legal pathway for experimental, ultra-rare disease treatments is rocky and expensive

Pharmalot Ed Silverman

STAT Plus: ‘We’ve exhausted all avenues’: A small biotech may give up on its ultra-rare disease drug over frustration with FDA

Morning Rounds Elizabeth Cooney

Morning Rounds: For kids with high BMI, task force not ready to recommend weight loss drugs

The Readout Damian Garde

STAT Plus: A plot to solve the ‘whack-a-mole’ of drug discovery

Special Report Brittany Trang

STAT Plus: Gene therapy offered this 7-year-old freedom. The price: a grueling year

The Readout Damian Garde

STAT Plus: Tome Biosciences goes public today in another biotech megaround

Pharmalot Ed Silverman

STAT Plus: Sanofi ends deal for a rare disease drug after FTC voices monopoly concerns

The Readout Theresa Gaffney

Morning Rounds: Preterm birth rates vary across the U.S., with the highest in the Southeast

Biotech Brittany Trang

STAT Plus: Tough road ahead for Bluebird Bio despite FDA approval for sickle cell therapy

The Readout Meghana Keshavan

CRISPR is about to make history. Here’s the 5-minute rundown of what to expect

Special Report Megan Molteni

New gene therapies confront many sickle cell patients with an impossible choice: a cure or fertility

Politics Sarah Owermohle

STAT Plus: Official: FDA needs staff influx to meet gene therapy needs

Morning Rounds Theresa Gaffney

STAT Plus: STAT Morning Rounds: Six health news stories you need to start your day

Biotech Jason Mast

STAT Plus: How sickle cell became the first disease treated by CRISPR

Biotech Damian Garde

STAT Plus: After four patients died of gene therapy, Astellas sees possible explanation — and a way forward

The Readout Meghana Keshavan

STAT Plus: The first vaccine for the increasingly prevalent chikungunya virus is here

First Opinion Gunnar Esiason

State Prescription Drug Affordability Boards could end up limiting access to the life-saving cystic fibrosis medication Trikafta

First Opinion Vivian G. Cheung

STAT Plus: How the government can help lower the price — not just the cost — of cutting-edge gene therapies

The Readout Meghana Keshavan

STAT Plus: Your Monday morning biotech news roundup

Politics John Wilkerson

STAT Plus: ALS patients support bill that could help patients with other rare diseases

Health Joanne Silberner

WHO considers adding ‘noma,’ a rare childhood disease, to its list of neglected conditions

The Readout Meghana Keshavan

STAT Plus: Why Biogen is purposefully becoming more risk averse, according to Biogen’s CEO

Biotech Jason Mast

STAT Plus: Peter Marks on creating Operation Warp Speed, but for rare diseases

The Readout Damian Garde

STAT Plus: With Ozempic, Wall Street moves on from a whole other class of medicines

Living With Isabella Cueto

With a rare immunodeficiency disease, a performer and patient advocate learned lessons the hard way