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Rare Daily Staff Genomics England has started to test hundreds of babies for more than 200 rare […]
Read moreShortening the Diagnostic Odyssey
Despite advancements in genetic testing, people with rare diseases often face a prolonged diagnostic odyssey involving multiple […]
Read moreCAMP4 Enters Collaboration with BioMarin to Advance Novel Regulatory RNA-Targeting Medicines
Rare Daily Staff CAMP4 Therapeutics said it entered into a research collaboration with BioMarin Pharmaceutical to advance […]
Read morePfizer Pulls Oxbrya from Market
Rare Daily Staff Pfizer said that it is voluntarily withdrawing all lots of Oxbryta for the treatment […]
Read moreMaking Exomes More Revealing
Standard exome sequencing, which maps the protein coding regions of the genome, will deliver a diagnosis of […]
Read moreBiohaven SCA Therapy Meets Primary Endpoint in Pivotal Study
Rare Daily Staff Biohaven a pivotal study of its experimental therapy troriluzole to treat the rare neurodegenerative […]
Read moreFDA Approves Zevra’s NPC Therapy
Rare Daily Staff The U.S. Food and Drug Administration approved Zevra Therapeutics Miplyffa, an oral therapy for […]
Read moreHow One Patient Organization Catalyzed Drug Development
Monica Coenraads, the mother of a daughter with Rett Syndrome, has played a critical role in catalyzing […]
Read moreAscendis Reports Positive Results for Experimental Achondroplasia Therapy
Rare Daily Staff Ascendis Pharma reported positive topline data from its pivotal double-blind placebo-controlled trial of its […]
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