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Rare Daily Staff The U.S. Food and Drug Administration has approved Pharming Group’s Joenja for the treatment […]
Read moreNHS Patients with rare genetic disorders to be fast-tracked to earlier diagnosis and specialist care
Rare Daily Staff NHS England said it is launching a new service to fast track diagnosis and […]
Read moreEuropean Commission Approves Amicus’ Pombiliti in Patients with Late-Onset Pompe Disease
Rare Daily Staff The European Commission has granted approval for Amicus Therapeutics’ Pombiliti, a long-term enzyme replacement […]
Read moreIonis and AstraZeneca Report Positive Topline 66-Week Results of Eplontersen Phase 3 Study for Patients with ATTRv-PN
Rare Daily Staff Ionis Pharmaceuticals and AstraZeneca reported positive topline results from the 66-week analysis of the […]
Read moreNICE Recommends PTC’s AADC Gene Therapy
Rare Daily Staff The United Kingdom’s National Institute for Health and Care Excellence, the drug price watchdog, […]
Read more2023 RARE Mental Health Grant Recipients Announced
Global Genes is pleased to announce the Rare Disease Patient Advocacy Organizations receiving 2023 RARE Mental Health […]
Read moreSeeking a Sustainable Business Developing on N-of-1 Therapies
The ability to pinpoint the underlying genetic causes of diseases and rapidly generate genetic medicines to address […]
Read moreGlobal Genes Team Shares Their Stories on World Rare Disease Day
My daughter Juno was diagnosed with a very rare, severe neurodevelopmental condition called STXBP1-related disorder six years […]
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