rare diseases

Pharmalot By Ed Silverman

Pharma seeks orphan designation for more drugs than ever before

Biotech By Meghana Keshavan

Q&A: How to mimic a brand-name drug — with one key substitution

Politics By Associated Press

Britain OKs making babies from DNA of three people in some cases

First Opinion By Nora Wong, Teneille Gofton and Sara Hocker

My son had a devastating, mysterious illness. The conversation about palliative care knocked me flat

First Opinion By Suzanne Koven

Why the election got me thinking about my patient’s painful struggle

Pharmalot By Ed Silverman

Obama extends controversial program for rare pediatric drugs

Health By Shayla Love

Is every disease a rare disease?

Pharmalot By Ed Silverman

Congress tries to fix a drug voucher program, but critics say it’s not enough

In the Lab By Karen Weintraub

Two newborn deaths in the same family set off a race to solve a medical mystery

Pharmalot By Ed Silverman

A family-run drug maker faces tough choices to stay afloat in the Shkreli era

Pharmalot By Ed Silverman

Pharmalot, Pharmalittle: Valeant doubled price on assisted-suicide drug

Pharmalot By Ed Silverman

FDA wants to nix voucher program for rare pediatric disease drugs

First Opinion By Christopher Anselmo

How a blood test changed my life and helped me become a rare disease advocate

Health By Andrew Joseph

Breaking down what the pope’s nod to birth control means for public health

Pharmalot By Ed Silverman

Canadian provinces close the door on Alexion over price of rare disease drug

Pharmalot By Ed Silverman

FDA designated a record number of orphan drugs last year

Politics By Ike Swetlitz

FDA urged to approve ‘three-parent embryos,’ a new frontier in reproduction

Pharmalot By Ed Silverman

Why the FDA shouldn’t bow to parental pressure over Duchenne drug

In the Lab By Karen Weintraub

A rare disease killed her mother. Can this scientist save herself?

Business By Dylan Scott

This biotech company is trying to make a rare disease drug cheaper. Will it work?

In the Lab By Rebecca Robbins

Big hopes for gene therapy dogged by uneven clinical results

Business By Andrew Joseph

Drug developers fund patient registries to collect data on rare diseases

Pharmalot By Ed Silverman

Bill creating more orphan drugs would cost taxpayers and patients

Health By Melissa Bailey

No longer invisible: The faces of rare disease

Pharmalot By Ed Silverman

Law for rare disease drugs needs revamping, researchers say

Business By Helen Branswell

How a system meant to develop drugs for rare diseases broke down

Health By Melissa Bailey

Boy’s mystery illness puts team of global scientists on the case

In the Lab By Helen Branswell

The strange case of the man infected with tapeworm cancer

Business By Rebecca Robbins

Funding plan targets drug development for rare diseases