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rare diseases

My son’s rare disease diagnosis spurred me to ditch Big Pharma for a biotech startup
Commentary
My son’s rare disease diagnosis spurred me to ditch Big Pharma for a biotech startup
By Michelle WernerMarch 10, 2025
Céline Dion says she may never tour again amid battle with stiff person syndrome. Here’s what to know
Health
Céline Dion says she may never tour again amid battle with stiff person syndrome. Here’s what to know
By Alexa Mikhail and Jordyn BradleyApril 23, 2024
‘Desperate for answers’: Thousands of people finally received diagnoses that had eluded them for years. Here’s how
Health
‘Desperate for answers’: Thousands of people finally received diagnoses that had eluded them for years. Here’s how
By Erin PraterApril 14, 2023
It takes an average of 8 years for a rare disease patient to get diagnosed. Why is it so hard to get life-altering genetic testing in the U.S.?
Health
It takes an average of 8 years for a rare disease patient to get diagnosed. Why is it so hard to get life-altering genetic testing in the U.S.?
By Erin PraterFebruary 28, 2023
Mother holds child's hand
Health
Parents are becoming drug developers to find a cure for their children’s rare diseases
By Jared Whitlock and Kaiser Health NewsJuly 21, 2022
Girl (8-9) smiling behind a pink protective face mask
Health
Long COVID in kids is worrying the experts: ‘It affects everybody differently and at different timescales’
By David MeyerOctober 20, 2021
Cures for many rare diseases might already exist. Why aren’t we using them?
Commentary
Cures for many rare diseases might already exist. Why aren’t we using them?
By Annette BakkerJuly 5, 2021
COVID forgotten communities
Newsletters
The communities we forget during COVID-19
By Sy MukherjeeApril 30, 2020
The coronavirus pandemic is impacting critical research into neuromuscular diseases
Health
The coronavirus pandemic is impacting critical research into neuromuscular diseases
By Sy MukherjeeApril 28, 2020
An FDA Program Incentivizing Rare Disease Drugs Will Be Investigated for Abuses
Health
An FDA Program Incentivizing Rare Disease Drugs Will Be Investigated for Abuses
By Sy MukherjeeMarch 22, 2017
Martin Shkreli’s Former Company, Back in Business, Vows No Crazy Drug Prices
Health
Martin Shkreli’s Former Company, Back in Business, Vows No Crazy Drug Prices
By Sy MukherjeeJuly 1, 2016
Shire Plc's U.K. Offices As Drugmaker Rejects Abbvie Inc.'s $46.5 Billion Offer
Shire switches from target to predator with $5.2 billion NPS buy
By Geoffrey SmithJanuary 12, 2015
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