Health Matthew Herper At a fashion show for people with spinal muscular atrophy, models take back their own stories
Exclusive Ed Silverman Novartis reports two children died from acute liver failure after treatment with Zolgensma gene therapy
A STAT Investigation Allison DeAngelis, Adam Feuerstein and Jason Mast At pioneering center for gene therapy, Jim Wilson presided over toxic, abusive workplace, staffers say
In the Lab Megan Molteni Provocative new findings suggest a surprising cause of Down syndrome: cells linked to aging
Health Damian Garde A new treatment promises to make little people taller. Is it an insult to ‘dwarf pride’?
First Opinion Nathan Yates I have spinal muscular atrophy. Critics of the $2 million new gene therapy are missing the point
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 Darcy Krueger and Emanual Maverakis The Access to Rare Indications Act could be a game changer for millions of Americans
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
In the Lab Eric Boodman This family carried a rare mutation that should have been lethal. What was keeping them alive?
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