rare diseases

Biotech Jonathan Wosen

STAT Plus: Mirum’s Chenodal succeeds in late-stage trial for rare metabolic disorder

Biotech Jason Mast

STAT Plus: Taysha Gene Therapies’ plan to tackle rare diseases is crumbling

First Opinion Rachel King and Peter L. Saltonstall

STAT Plus: The IRA needs changes to better support patients with rare diseases

Biotech Jonathan Wosen

STAT Plus: Neurocrine Biosciences drug for congenital adrenal hyperplasia succeeds in late-stage trial

Biotech Jonathan Wosen

STAT Plus: Crinetics acromegaly drug succeeds in late-stage trial but would face stiff competition if approved

Living With Isabella Cueto

An uncommon thyroid diagnosis led to an unexpected, all-too-visible side effect

Politics Rachel Cohrs Zhang

STAT Plus: AstraZeneca sues to stop Medicare drug price negotiation program

The Readout LOUD Damian Garde, Allison DeAngelis, and Adam Feuerstein

Listen: Racing for gene therapy, a pioneering approval, & startups in the lurch

Biotech Andrew Joseph and Damian Garde

STAT Plus: FDA approves first treatment for ultra-rare bone disease

Biotech Andrew Joseph and Damian Garde

STAT Plus: In the case of a devastating disease, the FDA weighs an experimental drug’s muddled data and a desperate need

Adam's Take Adam Feuerstein

STAT Plus: Anavex, maker of rare disease drug, keeps shifting the goalposts in its clinical trials

In the Lab Bree Iskandar

A young girl’s custom gene therapy hints at a framework for tailored rare disease treatments

First Opinion Annette Bakker

A seemingly small semantic issue is a major roadblock to develop treatments for rare diseases

Biotech Andrew Joseph

STAT Plus: FDA approves first treatment for skin condition that causes persistent wounds, a redosable gene therapy

First Opinion Emil D. Kakkis and Camille Bedrosian

Three misconceptions about the accelerated drug approval pathway

Biotech Jason Mast

STAT Plus: Gene therapy has helped boys like Conner walk. Scientists are trying to keep it that way

Biotech Jonathan Wosen

STAT Plus: Travere Therapeutics drug, approved for one rare and deadly kidney disease, fails in study of another

Health Andrew Joseph

Large European study underlines power of genomic sequencing to diagnose diseases

Pharmalot Ed Silverman

STAT Plus: Brazilian government advisory committee says a Vertex cystic fibrosis drug is not cost-effective and should not be reimbursed

Biotech Jonathan Saltzman — Boston Globe

STAT Plus: A young boy’s nightmare diagnosis, and the $3 million one-time treatment that will likely save his life

Health Andrew Joseph

STAT Plus: A devastating skin condition could soon get its first medication, as the frontiers of gene therapy expand

In the Lab Jason Mast

STAT Plus: Researchers devise new strategies to overcome a key CRISPR flaw

Biotech Jason Mast

STAT Plus: Top FDA official: Agency needs to start using accelerated approval for gene therapies

First Opinion Judy Stecker

My son’s time is running out due to a rare disease. The FDA needs to add more clinical trial flexibility

First Opinion Bruce Bloom

2022 was a breakthrough year for understanding rare diseases. 2023 needs to be better

Biotech Jonathan Saltzman — Boston Globe

STAT Plus: Gene therapy can transform life for people with hemophilia. But some patients don’t want it.

First Opinion Emil D. Kakkis

STAT Plus: As it turns 40, the Orphan Drug Act for rare diseases needs a refresh

In the Lab Megan Molteni

In a first, children with rare genetic diseases get mitochondrial transplants from their mothers

Biotech Matthew Herper and Jason Mast

STAT Plus: Amgen’s $27.8 billion Horizon merger is latest industry bet on pricey rare disease drugs