Neurodegenerative Diseases

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uniQure rockets on map to market for Huntington's therapy

uniQure says it has reached agreement with the FDA on a pathway to accelerated approval for its much-anticipated Huntington's disease gene therapy.

News

Woman pushing man in wheelchair along beachfront as sun sets

Novartis pays $1bn upfront for Huntington's drug from PTC

Novartis has made a return to the Huntington's disease stage by licensing an oral mRNA splicer from PTC Therapeutics for $1 billion upfront.

News

uniQure rockets on Huntington’s gene therapy result

Shares in gene therapy developer uniQure have risen more than 75% after the company reported that Huntington’s disease candidate AMT-130 slowed down the progression of the disorder over two

News

NeuroSense says ALS drug extends survival

NeuroSense Therapeutics is currently facing de-listing from the Nasdaq, so encouraging data from a phase 2b trial of its PrimeC drug candidate for amyotrophic lateral sclerosis (ALS) comes

R&D

Purity, potency, and safety – affordably – in stem cell ther...

In a new episode of the pharmaphorum podcast, web editor Nicole Raleigh discusses stem cell therapy, and accessibility to and affordability of s

News

Sage Therapeutics' chief executive Barry Greene

Sage drops dalzanemdor for Parkinson’s after trial flop

Sage Therapeutics has suffered another disappointment after deciding that it will have to drop the Parkinson’s programme for key pipeline drug dalzanemdor after it showed

uniQure rockets on map to market for Huntington's therapy

Novartis pays $1bn upfront for Huntington's drug from PTC

uniQure rockets on Huntington’s gene therapy result

NeuroSense says ALS drug extends survival

Purity, potency, and safety – affordably – in stem cell ther...

Sage drops dalzanemdor for Parkinson’s after trial flop

Daiichi and AZ pull Dato-DXd filing in Europe too

The AI effect in pharma commercial strategy: From data to re...

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