Featured
When a Miracle Cure Is Left on the Shelf
When a Miracle Cure Is Left on the Shelf
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Gene Therapy Allows an 11-Year-Old Boy to Hear for the First Time
Gene Therapy Allows an 11-Year-Old Boy to Hear for the First Time
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Alpha Rev's Casey McPherson Gave Up a Life in Music to Cure His Daughter's Rare Disease
Alpha Rev's Casey McPherson Gave Up a Life in Music to Cure His Daughter's Rare Disease
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These twins share everything including a rare disease and surgery on the same day
These twins share everything including a rare disease and surgery on the same day
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A young girl’s custom gene therapy hints at a framework for tailored rare disease treatments
A young girl’s custom gene therapy hints at a framework for tailored rare disease treatments
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A young boy’s nightmare diagnosis, and the $3 million one-time treatment that will likely save his life
A young boy’s nightmare diagnosis, and the $3 million one-time treatment that will likely save his life
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Gene therapy trial shows promise for treating ‘bubble boy’ syndrome. Now comes the hard part
Gene therapy trial shows promise for treating ‘bubble boy’ syndrome. Now comes the hard part
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Revenge of the gaslit patients
Revenge of the gaslit patients
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Like Celine Dion, Pennsylvania man is fighting Stiff Person Syndrome 'with everything I have’
Like Celine Dion, Pennsylvania man is fighting Stiff Person Syndrome 'with everything I have’
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FDA needs to be more flexible assessing rare
FDA needs to be more flexible assessing rare
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How medicine erased Black women from a "White Man's Disease"
How medicine erased Black women from a "White Man's Disease"
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The parents hoped an existing drug might keep their kids from having seizures. Then they saw the price
The parents hoped an existing drug might keep their kids from having seizures. Then they saw the price
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ALS groups to the FDA and drug sponsors: ‘We won’t be played again’
ALS groups to the FDA and drug sponsors: ‘We won’t be played again’
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The Real Work of Parenting a Rare Girl
The Real Work of Parenting a Rare Girl
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NY Times: It will consume your life: 4 families take on rare diseases
NY Times: It will consume your life: 4 families take on rare diseases
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Rare in Common Interview
Rare in Common Interview
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Texas mom helps local researchers find treatments for genetics causes of autism
Texas mom helps local researchers find treatments for genetics causes of autism
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Saving Mila: How a tailor-made therapy, developed in a flash, may have halted a young girl’s rare disease
Saving Mila: How a tailor-made therapy, developed in a flash, may have halted a young girl’s rare disease
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Richard Engel: Our son’s special needs bring heartbreak, but we have hope – Today Show
Richard Engel: Our son’s special needs bring heartbreak, but we have hope – Today Show
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Sophie’s search for a cure – multiple system atrophy – You Tube
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