ScienceDaily (Mar. 9, 2010) — They are considered to be the most important model organism for research into human biology: mice may look totally different, but they are in many ways similar to Homo sapiens on a fundamental level. For instance, an impressive 99 per cent of the mouse genes are matched by a corresponding sequence in the human genome. That is also why the law in this part of the world only permits scientists to conduct research on human embryo stem cells when they have "clarified in advance" their specific questions by using animal cells as far as possible. However, such tests are often pointless -- and sometimes even misleading, as a recent study by scientists working with Hans Schöler at the Max Planck Institute for Molecular Biomedicine in Münster demonstrates.

Penwest Pharmaceuticals Co. (PPCO)
Q4 2009 Earnings Call

March 4, 2010 11:00 am ET

(…) The first study which we initiated in December 2009 is in patients with Friedreich's Ataxia or FA. FA which afflicts one in 50,000 people in the US is a debilitating, life shortening, degenerative neuromuscular disorder. FA patients have gene mutations that limit the production of a protein call frataxin, an important protein that functions in the (Technical Difficulty) – and thus affects energy function. Onset of symptoms can vary from childhood to adulthood.

Mark Henderson / The Times

March 1, 2010 -  A new treatment for an incurable wasting disease that kills about 50 British infants every year could begin human trials within two years, after highly encouraging results in animals.
The gene therapy for spinal muscular atrophy (SMA), which aims to correct the faulty DNA that causes the condition, has dramatically prolonged the lives of mice with the mutation, while improving their nerve and muscle function. The treated rodents lived for more than 250 days, which is five times longer than those given any other type of therapy and 16 times longer than untreated animals.