rare diseases

Biotech Jason Mast

STAT Plus: Can AI build custom ‘n-of-1’ drugs faster? Maybe not yet, new case study suggests

Biotech Andrew Joseph

STAT Plus: After its last deal fell through, Maze Therapeutics finds new partner for rare disease drug

In the Lab Andrew Joseph

STAT Plus: Experimental gene therapy seems to alleviate skeletal defects tied to rare inherited disease, study shows

Health Andrew Joseph

STAT Plus: Why a British hospital, and not a drugmaker, is trying to get a rare disease therapy approved

In the Lab Megan Molteni

STAT Plus: In a scientific first, researchers use CRISPR base editing to treat liver disease in fetal monkeys

Biotech Allison DeAngelis

STAT Plus: Ultragenyx reports early success with Angelman syndrome drug, backing up 2022 acquisition

Pharmalot Ed Silverman

STAT Plus: FDA agrees to review a rare disease drug that its developer was about to give up on

The Readout Meghana Keshavan

STAT Plus: How resurrecting extinct species might impact medicine

Biotech Andrew Joseph

STAT Plus: Early data from rare-disease trial put spotlight on Moderna’s efforts to turn mRNA into medicines

Biotech Andrew Joseph

STAT Plus: Critics say U.K. decision on ALS drug could have ‘chilling effect’ on access to new genetic medicines

In the Lab Andrew Joseph

STAT Plus: U.K. health officials and researchers try to carve a path for more individualized genetic treatments

Biotech Jason Mast

STAT Plus: FDA approves first gene therapy for fatal neuron disease in children

Pharma Andrew Joseph

STAT Plus: AstraZeneca picks up rare disease-focused Amolyt Pharma in $1 billion deal

Health Jonathan Saltzman — Boston Globe

STAT Plus: He’s 19 months old and has one of the world’s rarest diseases. His parents are determined to find a cure.

The Readout Damian Garde

STAT Plus: The FDA isn’t afraid to yank a disappointing drug

Exclusive Megan Molteni

Texas Medicaid agrees to fully cover gene therapy for Afghan refugees’ infant

Biotech Jason Mast

STAT Plus: Obscure vertigo drug could bring rare hope for patients with devastating neuron disease

Health Megan Molteni

In partial reversal, Texas Medicaid greenlights first step in gene therapy for Afghan refugees’ infant son

Exclusive Megan Molteni

STAT Plus: The Afghan father aided U.S. soldiers. Now Texas Medicaid won’t pay for his son’s gene therapy

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