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In Rare Form

A total of 29 posts are filed under In Rare Form
Are Orphan Drugs Losing their Luster?
The orphan drug market is experiencing strong growth, but there are signs that pricing pressures may be impacting the sector, raising… Continue Reading
Anarchy in the U.K.
Rare disease patients in the United Kingdom face unusual barriers to accessing medicines because of the multiple review and reimbursement… Continue Reading
A Victory for One
In 1886, the physician Jonathan Hutchinson examined a six-year-old patient who lacked hair and had the withered skin of an old man. Later,… Continue Reading
Why Patient Groups Shouldn’t Dismiss Report on Pharma Influence
Earlier this month, Kaiser Health News launched “Pre$cription for Power,” a database that tracks Big Pharma contributions to patient… Continue Reading
How Maeve Sheehy Learned to Dream
Maeve Sheehy fell asleep as a teenager and woke up as an adult. Sheehy began nodding off in classes at the age of 13 when she started middle… Continue Reading
Why Rare Diseases May Be More Common Than Thought
Rare genetic diseases can be difficult for doctors to recognize, particularly when the symptoms of a disease fall short of dramatic,… Continue Reading
A Matchmaker for Patients Seeking Clinical Trials
In the past day, I’ve had several rare and common diseases, including some horrendous stuff, but all in the name of science. My sudden… Continue Reading
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