U.S. health officials have added two deadly rare diseases to the list of conditions the government recommends states screen ...
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Shionogi buys global RADICAVA rights in $2.5 billion ALS drug deal to build rare disease platform
An announcement from Shionogi & Co ( ($JP:4507) ) is now available. Shionogi has agreed to acquire from Tanabe Pharma the global rights and assets ...
The energy that powers cells of the human body comes from tiny cellular components called mitochondria. Just as a draining battery makes it difficult to operate an electronic device, mitochondrial ...
Ionis Pharmaceuticals is eyeing a new rare disease approval after its antisense oligonucleotide zilganersen successfully improved mobility in patients with the neurological condition Alexander disease ...
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BioMarin to buy rare disease drugmaker Amicus Therapeutics for $4.8B
BioMarin Pharmaceutical BMRN announced that it entered a definitive agreement to acquire all outstanding shares of Amicus ...
Catherine Illingworth, 39, knew something was wrong the moment her son's neurologist walked into the room. Illingworth requested the referral from her son's pediatrician based on instinct. George took ...
A phase 2 study of Vigil Neuroscience’s iluzanebart has missed its biomarker and efficacy endpoints, prompting the Sanofi-bound biotech to stop a long-term extension trial of the TREM2 agonist. Sanofi ...
Baylor College of Medicine has received new funding from the National Institutes of Health for two consortia in the Rare Diseases Clinical Research Network: $7.4 million over five years to renew the ...
When Casey McPherson became a father, his purpose became clear. He needed to raise his daughter, Rose, to be loving, courageous, healthy and strong. He needed to protect her. Many parents can ...
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