Gene Therapy News

Gene therapy restarts patients’ heart stem cells

In a breakthrough clinical trial on humans, researchers used gene therapy to encourage the heart to repair itself after cardiac damage.

Mounting evidence has pointed towards the heart having a regenerative potential but its capacity for regeneration is insufficient to replace the massive loss of cardiomyocytes following ischaemic damage.

Experimental Treatment For DMD Offers Hope for Youngster

SACRAMENTO, California, USA. Jacob Rutt is a bright 11-year-old who likes to draw detailed maps in his spare time. But the budding geographer has a hard time with physical skills most children take for granted ― running and climbing trees are beyond him, and even walking can be difficult. He was diagnosed with a form of muscular dystrophy known as Duchenne when he was two years old.

uniQure's gene therapy Glybera® recommended for approval

AMSTERDAM, The Netherlands, July 20, 2012

uniQure announced today that the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion that recommends marketing authorization of Glybera® (alipogene tiparvovec) as a treatment for lipoprotein lipase deficiency (LPLD) under exceptional circumstances. LPLD is a very rare, inherited disease. Patients with the disease are unable to handle fat particles in their blood plasma, which leads to recurring severe abdominal pain and pancreatitis.