March 23, 2018
A Task for Tyler- Finding Treatments for SSADH and Hypotonia
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March 19, 2018
2q23.1 Diagnosis Leads Mom to Start Online Boutique for Daughter’s Future
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March 15, 2018
Ionis and Akcea Form hATTR Partnership Worth up to $1.7 Billion
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March 15, 2018
Flagship Pioneering Backs Foghorn Therapeutics with $50 Million to Develop New Class of…
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March 12, 2018
Sanofi Passes on Alnylam Primary Hyperoxaluria Type 1 Candidate
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March 12, 2018
Sarepta Therapeutics Plans to Submit DMD Drug Golodirsen for FDA Approval by Year…
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March 9, 2018
Don’t Forget About the Adults
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March 8, 2018
Rare Leader: Lale’ Welsh, CEO of the Neuromuscular Disease Foundation
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March 7, 2018
5 Things I’ve Learned About Living With a Rare Disease
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March 6, 2018
Proposed RARE Act Seeks to Address Common Challenges Rare Disease Patients Face
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March 1, 2018
The Benefits of Patient-to Patient Interactions
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