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Gene Therapy Receives Orphan Drug and Rare Pediatric Disease Designations

Gene Therapy Receives Orphan Drug and Rare Pediatric Disease Designations
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National-center
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Journal-of-the-european-society-gene
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Orphan-drug-designation
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BioSpace Movers & Shakers, March 4

Biopharma and life sciences companies strengthen their leadership teams and boards with these Movers & Shakers.

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Alltrna Appoints Rare Disease Clinical Research Expert, Dr. Anne Pariser, as Vice President, Medical and Regulatory Affairs

/PRNewswire/ Alltrna, a Flagship Pioneering company unlocking transfer RNA (tRNA) biology and pioneering tRNA therapeutics to regulate the protein universe.

Cambridge
Cambridgeshire
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8 things medical TV shows get wrong

8 things medical TV shows get wrong
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Diagnostic odyssey: The lonely road walked by thousands of Coloradans with rare disorders

When Keegan Joines was born as a low-birthweight baby, his parents saw the rough start as a mere bump in the road.

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