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 ...
Tag: Rare
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 ...
Roche suffers a fresh blow as India’s Supreme Court allows generic sales of its pricey rare disease drug
In a case that was billed as profits versus patients, India’s Supreme Court dismissed a petition in which Roche sought ...
Sarepta to seek approval for gene therapy in rare muscular dystrophy
An experimental gene therapy from Sarepta Therapeutics increased levels of the gene missing in an ultra-rare form of muscular dystrophy, ...
Chiesi’s Rare Disease Strategy Expands to Genetic Meds via Alliance With CRISPR Startup Arbor
In the five years since Italy-based Chiesi Group established its rare disease division in Boston, the unit has landed regulatory ...
With FDA Nod in Rare Hormone Disease, Crinetics Can Challenge Blockbuster Meds From Novartis, Ipsen
Drugs for the rare hormone disorder acromegaly include older engineered peptides administered as frequent injections. A new FDA approval brings ...
CDC: Rare Salmonella strain from bearded dragons caused 2024 US outbreak, still circulates
Kent Kanouse / Flickr cc People who live with or handle pet bearded dragons are at continued risk for Salmonella ...
First Therapy for Rare Tumor Is a Blockbuster Prospect, But Does Precigen Have the Cash to Make It Happen?
Tumors caused by recurrent respiratory papillomatosis are benign, but that doesn’t mean they are not serious. In this rare disease, ...
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