rare disease

Biotech By Elizabeth Cooney

Wary hemophilia patients say they’re willing to wait longer for a safe gene therapy

Biotech By Adam Feuerstein and Damian Garde

Stunning FDA rejection signals potential new hurdles for gene therapies

Pharmalot By Ed Silverman

A long-awaited report on orphan drugs in Europe suggests incentives to pharma need change

In the Lab By Sharon Begley

Gene-editing discovery could point the way toward a ‘holy grail’: cures for mitochondrial diseases

First Opinion By Michelle McMurry-Heath

A pandemic, a funeral, and a chance to help heal the world

First Opinion By Christopher Anselmo

I’ve been cared for by dozens of clinicians in nearly 20 years. I’m in awe of their work on Covid-19

Pharmalot By Ed Silverman

FDA closes review of Novartis data integrity scandal without punishing the drug maker

Biotech By Damian Garde

To quell unpredictable allergic reactions, an experimental drug takes aim at a genetic cause, not symptoms

Pharmalot By Ed Silverman

Oh, Canada! Vertex battles another country over access to cystic fibrosis treatments

First Opinion By Ethan J. Weiss

Should ‘broken’ genes be fixed? My daughter changed the way I think about that question

Pharmalot By Ed Silverman

Pharma execs criticize a drug maker over pricing and claim orphan drug law is being misused

Off the Charts By Jennifer Adaeze Okwerekwu

As medical professionals, my husband and I know the Nigerian travel ban is cruel. Now the pain is personal

Health By Andrew Joseph and Ed Silverman

A lottery like no other offers up a cutting-edge medicine — with lives on the line

Adam's Take By Adam Feuerstein

If biotech had a Mount Rushmore, whose heads would you chisel in the granite?

Health By Rebecca Robbins

Chan Zuckerberg Initiative to fund 30 patient groups, aiming to build a model for tackling rare diseases

Biotech By Matthew Herper, Adam Feuerstein, and Damian Garde

What does the latest twist in the Sarepta saga mean for the future of the FDA? We discuss

Biotech By Adam Feuerstein

On the cusp of FDA approval for its NASH drug, Intercept’s CEO talks payers, pruritis, and price

Biotech By Jonathan Saltzman — Boston Globe

Blueprint gains approval for its first drug, a treatment for a rare gastrointestinal cancer

Biotech By Damian Garde

Regeneron drug reduces bone growth for patients with ultra-rare skeletal disease

Pharmalot By Ed Silverman

As families in Europe clamor for its pricey gene therapy, Novartis creates a lottery

Biotech By Jonathan Saltzman — Boston Globe

Ring Therapeutics is developing a new way to deliver gene therapy

Health By Eric Boodman

Waning treatment for immune deficiency is a warning for all ‘one-and-done’ therapies

Biotech By Jonathan Saltzman — Boston Globe

Startup targeting DNA repeats in Huntington’s and other diseases debuts with $59 million

Biotech By Kate Sheridan

Alnylam chalks up another win with phase 3 trial of a rare-disease drug

Biotech By Damian Garde

FDA changes course on Sarepta’s once-rejected muscular dystrophy drug