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Regulatory

A total of 39 posts are filed under Regulatory
U.S. Food and Drug Administration Orphan Drug Designations in March 2018
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MHRA Grants FCS Patients in UK Early Access to Akcea’s Volanesorsen
Rare Daily Staff The U.K.’s Medicines & Healthcare products Regulatory Agency said it would allow patients with the rare genetic lipid… Continue Reading
Giving Voice to Patients at the FDA
  Nearly a year ago, more than 400 members of spinal muscular atrophy community along with industry, clinicians, and researchers convened a… Continue Reading
Gottlieb Highlights New FDA Rare Disease Efforts to Address Rare Disease Challenges
Rare Daily Staff U.S. Food and Drug Administration Commissioner Scott Gottlieb in a blog entry today outlined several steps the agency is… Continue Reading
Gottlieb Calls for $400 Million in Expanded FDA Funding in Part to Stimulate Investment in Rare Diseases
Rare Daily Staff U.S. Food and Drug Administration Commissioner Scott Gottlieb called for developing clinical trial networks to improve the… Continue Reading
Patient Groups Call on House to Reject Right to Try Act
Rare Daily Staff The National Organization for Rare Disorders along with 37 patient and provider organizations in a letter to leaders of the… Continue Reading
U.S. Food and Drug Administration Orphan Drug Designations in January 2018
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