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Rare Daily Staff NHS England said it is launching a new service to fast track diagnosis and […]
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Rare Daily Staff The United Kingdom’s National Institute for Health and Care Excellence, the drug price watchdog, […]
Read moreHow Data Sharing Can Improve Diagnosis and Treatment for Rare Diseases
Study Makes Economic Case for Benefits of Data Sharing to Improve Diagnosis and Treatment of Rare Disease […]
Read moreHow One Rare Disease Advocate Learned to Look Across Diseases
by Danny Levine When RARE-X first sought to recruit Megan O’Boyle as patient engagement lead for the […]
Read moreRadically Changing Data Collection and Accelerating Advances in Rare Disease
RARE-X unveils plan to radically change data collection and sharing to accelerate advances for patients in rare […]
Read moreSenate Pressure Grows on Novartis over Gene Therapy Data Manipulation
Rare Daily Staff Senate Finance Committee Chairman Chuck Grassley (R-Iowa) is demanding records and answers from Novartis […]
Read moreSarepta Says DMD Gene Therapy Adverse Event Report Submitted Erroneously
Rare Daily Staff Sarepta Therapeutics said that it was informed that an adverse event report regarding its […]
Read moreFDA Updates Guidance on Rare Pediatric Disease Priority Review Vouchers
Rare Daily Staff The U.S. Food and Drug Administration issued updated draft guidance on Rare Pediatric Disease […]
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