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Rare Daily Staff Astellas Pharma has secured an exclusive option and license agreement for AVB-101, AviadoBio’s experimental […]
Read moreJudo Bio Launches with $100 Million to Develop Oligonucleotide Therapies that Target the Kidney
Rare Daily Staff Judo Bio, which is developing oligonucleotide medicines delivered to the kidney, said it raised […]
Read moreGenomics England Screens First Newborns for More Than 200 Genetic Conditions as New Study Begins
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 […]
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