A novel gene therapy technique is safe and may be effective at staving off worsening symptoms of Parkinson's disease, according to the first scientific review of a dozen patients who have received the treatment over the last three years. The results were published in the latest issue of Lancet. The patients, half of whom live on Long Island, are in advanced stages of the illness and were no longer responding to medicines when they signed on for the experimental therapy. [click link for full article]

The Food and Drug Administration has announced the approval of Carnitor(R) SF (levocarnitine) Sugar-Free Oral Solution for the same indication approved for the current Carnitor(R) Oral Solution containing sugar. Carnitor(R) SF and Carnitor(R) Oral Solution are indicated to treat primary systemic carnitine deficiency and for acute and chronic treatment of patients with an inborn error of metabolism which results in a secondary carnitine deficiency. [click link for full article]

Families of Spinal Muscular Atrophy (FSMA) is pleased to announce the selection of a Clinical Candidate for Spinal Muscular Atrophy through its program being conducted at deCODE chemistry. At the same time FSMA is now extending its contract with deCODE to continue work towards an Investigational New Drug (IND) application with the Food and Drug Administration. If successful, this would be the first novel drug designed specifically to treat Spinal Muscular Atrophy (SMA). [click link for full article]

Gene expression oscillates throughout the day, suggesting that interfering with the body's circadian rhythms could have profound affects

Injecting a gene into the brains of people suffering from Parkinson's disease reduced symptoms by as much as 70%, researchers say

Our bodies could not maintain their existence without thousands of proteins performing myriad vital tasks within cells. Since malfunctioning proteins can cause disease, the study of protein structure and function can lead to the development of drugs and treatments for numerous disorders. For example, the discovery of insulin's role in diabetes paved the way for the development of a treatment based on insulin injections. [click link for full article]

Investigators at St. Jude Children's Research Hospital have shown that when the cancer drug irinotecan is given in low doses for multiple days, it eliminates the need to delay treatment to perform costly genetic testing that determines if the patient is at risk for serious treatment side effects, such as neutropenia. Neutropenia is an abnormal reduction in the numbers of immune cells, called neutrophils; the disorder leaves individuals more vulnerable to infections. [click link for full article]

Injection of genetic information directly into the brain cells of patients (gene therapy) with neurodegenerative conditions such as Parkinson's disease could safely alleviate symptoms of these conditions, conclude authors of an Article published in this week's edition of The Lancet. However an accompanying comment questions the advantages of gene therapy over deep-brain stimulation, a current method for treating Parkinson's disease. [click link for full article]

In what could be a breakthrough in the treatment of neurological disease, a team led by physician-scientists at NewYork-Presbyterian Hospital/Weill Cornell Medical Center has completed the first-ever phase 1 clinical trial using gene therapy to battle Parkinson's disease. [click link for full article]