Search

European regulators have approved the first therapy in the western world that can correct errors in a person’s genetic code, according to Amsterdam-based uniQure (formerly Amsterdam Molecular Therapeutics).

Europe has approved Glybera for treatment of Lipoprotein Lipase Deficiency (LPLD), a very rare, inherited disease. Patients with LPLD are unable to metabolize the fat particles carried in their blood, which leads to inflammation of the pancreas (pancreatitis).

Glybera introduces a normal, healthy LPL gene into the body so that it can make functional LPL protein. The LPL gene is packaged in a delivery vector derived from adeno-associated virus (AAV), serotype 1, which has a natural propensity towards muscle cells.

Glybera will be available in the second half of 2013, according to uniQure, which is preparing to apply for regulatory approval in the U.S., Canada, and other markets.

Source: http://www.kurzweilai.net/first-therapy-in-the-western-world-to-correct-errors-in-a-persons-genetic-code-approved



I want to help out, but I don’t know how

Have you ever wanted to help out the MSUD Family Support Group but were unsure about how to contribute? The MSUD Family Support Group is actively looking for volunteers to assist with advocacy and fundraising efforts.

Read More

MSUD Awareness Day

Now more than ever before we are hearing about National Awareness Days which are set by organizations or the government to bring attention to issues of importance.

Read More

Galen Carrington Classic MSUD age 27

Galen Carrington is a 27 year old Indiana resident who has classic MSUD. He underwent liver transplantation 5 years ago at Children’s Hospital

Read More

Fundraising

In the 2016 survey of the membership of the MSUD Family Support Group, research for improved treatments and potential cure was rated “most important” by 90% of the responding members.

Read More

MSUD Advocacy Update

Healthcare and issues facing the rare disease community have been at the forefront of national conversations over the past 6 months.

Read More

20th Annual White Horse Beach Charity Golf Tournament

On July 29, 2017 Charlie O’Rouke and his committee held the 20th Annual White Horse Beach Charity Golf Tournament.

Read More

Transform

A Child's Life

Subscribe to our mailing list

Signup To Our Newsletter Signup with your email address to receive news and updates