Boosting communication between the spinal nerves and the muscles using the spinal cord stimulation reverses spinal muscle atrophy (SMA) progression and could be applied to other motoneuron diseases, ...

The oversubscribed Series B will boost the clinical development of two RNAi therapies for epilepsy and Huntington’s disease ...

LEXINGTON, Mass. - uniQure N.V. (NASDAQ: NASDAQ:QURE), a gene therapy company with a market capitalization of $767 million, ...

A new drug-free, minimally invasive intervention targets the root cause of progressive loss of neural function in spinal muscle atrophy (SMA), an inherited neuromuscular disease.

PTC Therapeutics, Inc. (NASDAQ:PTCT), a biopharmaceutical company focused on developing and commercializing novel medicines for rare disorders, finds itself at a critical juncture. With a promising ...

Novartis has made a dramatic return to the Huntington's disease therapy stage by licensing a candidate from PTC Therapeutics for a whopping $1 billion upfront. The deal – which has a potential ...

In a small human trial in SMA patients neurostimulation sessions improved motoneuron function, reduced fatigue, and improved leg muscle strength and walking.

Shares of uniQure (NASDAQ:QURE – Get Free Report) have received a consensus rating of “Buy” from the ten ratings firms that ...

A new drug-free, minimally invasive intervention targets the root cause of progressive loss of neural function in spinal ...

Leerink Partners analyst Joseph Schwartz has reiterated their bullish stance on QURE stock, giving a Buy rating on January 27.Maximize Your ...

Faced with the encroaching threats of patent expirations and generics, biopharma companies in 2024 invested 33% more in ...

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 years.