rare disease

Health Ambar Castillo

‘All the tech in the world doesn’t solve this’: Rare disease experts push biopharma on equity

In the Lab Megan Molteni

On the long road to treating Huntington’s genetic stutter, scientists return to overlooked clues

Biotech Ryan Cross — The Boston Globe

Muscular dystrophy patient who was first in line for a custom CRISPR therapy dies

Politics Ambar Castillo

House Democrat presses bill to encourage more diversity in clinical trials run by NIH

First Opinion Karen Quandt

The FDA needs to be more flexible in assessing treatments for rare diseases, like the one that seemed to help my son

Biotech Matthew Herper

Blueprint Medicines drug reduces mastocytosis symptoms in a new study, meeting investor expectations

First Opinion Irene Shapiro

The Morris ALS Principles: A model for empowering all disease communities

Pharmalot Ed Silverman

‘No magic bullet’: For drugmakers and the FDA, clinical trials on ultra-rare diseases pose thorny challenges

First Opinion Craig Martin

There’s no ‘Moonshot’ or ‘Warp Speed’ for rare diseases. There should be

Biotech Damian Garde

FDA advisers endorse Bluebird gene therapy for rare blood disorder

Biotech Damian Garde

FDA advisers unanimously endorse Bluebird’s gene therapy for rare disorder

First Opinion Darcy Krueger and Emanual Maverakis

The Access to Rare Indications Act could be a game changer for millions of Americans

In the Lab Andrew Joseph

Child’s autopsy from 2008 becomes a clue to mystifying polio-like illness

First Opinion Brian Wallach and Mike Braun

Take a stand for those who can’t with the Promising Pathway Act

First Opinion Walter G. Johnson and Diana M. Bowman

It’s time for Congress to stop blocking mitochondrial replacement therapy

First Opinion Bernard Zipprich

Will the shadow of Aduhelm cloud FDA’s decision-making on an ALS therapy?

Pharmalot Ed Silverman

A $2.1 million price tag for Bluebird Bio’s gene therapy is cost-effective, analysis finds

Pharmalot Elizabeth Cooney

Pharmalittle: Advisory panel narrowly votes down ALS drug; Vertex looks to advance experimental pain drug

Video Chat Matthew Herper

Virtual Event: CRISPR Quest

In the Lab Eric Boodman

This family carried a rare mutation that should have been lethal. What was keeping them alive?

Health Jared Whitlock

A mother, shaped by tragedy, embarks on a mission to advance custom medicines

Health Jonathan Wosen

Researchers shatter the speed record for diagnosing rare genetic diseases with DNA sequencing

In the Lab Megan Molteni

Last-gasp gene therapy saved a Syrian refugee’s life. Five years later, the boy is thriving and a clinical trial is starting

Biotech Matthew Herper

Acadia drug meets endpoints for treating Rett syndrome, a rare neurological disease

Health Andrew Joseph

Sequencing whole genomes helps diagnose far more rare diseases, study shows