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Frontotemporal dementia: the state of treatment development

Pharmaceutical Technology

Other lines of research look at the genetic overlap between FTD and amyotrophic lateral sclerosis (ALS), which could be used in the development of treatments for both conditions. This is also one of the major challenges that slow the development of effective treatments for FTD, he adds. This protein promotes lysosomal function.

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Novo Nordisk’s gene silencing alliance with Dicerna bears first fruit

pharmaphorum

Two years after starting to work together, Novo Nordisk and Dicerna have selected the first candidate from a joint project to find new, gene-silencing drugs for liver-related cardiometabolic diseases. The nomination of a first candidate in the collaboration that will advance into development triggers a $2.5

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Study resolves long-running controversy over critical step in gene silencing

Scienmag

The findings could yield important implications for development of drugs to treat cancer and other diseases. BOSTON – A long-running debate over how an important gene-silencing protein identifies its targets has been resolved by researchers at Massachusetts General Hospital (MGH).

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Novo Nordisk snaps up RNAi partner Dicerna in $3.3bn takeover deal

pharmaphorum

Danish drugmaker Novo Nordisk must like what it has seen in its two-year-old alliance with gene-silencing specialist Dicerna Pharma – it has just agreed to acquire the biotech for $3.3 Once in the blood, the GalXC molecules travel to the liver, where they enter hepatocyte cells and can switch protein production on or off.

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NICE backs Alnylam’s Amvuttra for rare form of amyloidosis

pharmaphorum

Alnylam’s gene-silencing drug Amvuttra has been recommended as a treatment for hereditary transthyretin-related (ATTR) amyloidosis by NICE, paving the way for the drug to be made available on the NHS in England and Wales.

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REGENERON AND ALNYLAM REPORT POSITIVE INTERIM PHASE 1 

The Pharma Data

(Nasdaq: ALNY ) announced today positive interim results from the ongoing single ascending dose part of the Phase 1 study of ALN-APP, an investigational RNAi therapeutic targeting amyloid precursor protein (APP) in development for the treatment of Alzheimer’s disease and cerebral amyloid angiopathy (CAA). Yancopoulos, M.D.,

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Novo Nordisk bulks up in rare diseases with $1.2bn Prothena deal

pharmaphorum

The bulk of Novo Nordisk’s revenues come from its diabetes products, although it also active in the development and sale of drugs for growth disorders, haemophilia and sickle cell disease. In ATTR amyloidosis, transthyretin (TTR) protein misfolds and accumulates as amyloid deposits throughout the body.