Atrium Therapeutics Launches With $270M for RNA Therapies Addressing Rare Cardio Conditions
Atrium spun out of Avidity Biosciences, which Novartis acquired in a $12 billion deal. The Atrium pipeline is led by ...
Tag: Rare
FDA unveils new pathway for rare disease treatments
The Trump Administration on Monday launched detailed steerage for approving the primary bespoke medicines crafted to deal with sufferers’ particular person ...
Disc Medicine Rare Disease Drug Picked for Faster Regulatory Review Is Rejected by the FDA
Disc Medication’s effort to introduce a brand new remedy for a uncommon blood dysfunction with few remedy choices has been ...
PTC Therapeutics Ends Bid to Seek FDA Approval for Rare Muscle Disease Drug
For greater than a decade, PTC Therapeutics has been going backwards and forwards with the FDA to discover a path ...
How a Rare Elbow Megaprosthesis Restored a Life After 27 Years
The trigger traced again to a extreme elbow harm in his youth, one which steadily reshaped each bone and every ...
Review of 200 novel human viruses over a century a reminder that pathogen emergence isn’t rare
A systematic review of more than 200 studies published in BMC Infectious Diseases on human viruses over more than a ...
BioMarin’s Presence in Rare Enzyme Disorders Grows With $4.8B Amicus Therapeutics Acquisition
Rare disease drug developer BioMarin Pharmaceutical is buying its way to revenue growth with a $4.8 billion deal for Amicus ...
BioMarin to buy rare disease focused Amicus for $4.8B
BioMarin said Friday it would purchase Amicus Therapeutics for $4.8 billion, merging two rare disease-focused biotechs founded around the turn ...
Gene Therapy for Children With Rare ‘Bubble Boy Disease’ Proves Effective After Long-Term Follow-Up
The 62 children who were found to have the rare "Bubble Boy disease" as babies and toddlers between 2012 and ...
Liver Complications Lead FDA to Stop Tests of Intellia Gene-Editing Therapy for a Rare Disease
The FDA has halted clinical testing of an Intellia Therapeutics gene-editing therapy following a report that a patient in a ...
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