Rare Disease News and Research

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Muscadine grape skin extract shows promise for prostate cancer

Laboratory experiments show that an extract of the skin of muscadine grapes can inhibit growth of prostate cancer cells in the laboratory.

4 Sep 2007

Fruit flies have a taste for carbonated water

That fruit fly hovering over your kitchen counter may be attracted to more than the bananas that are going brown; it may also want a sip of your carbonated water.

4 Sep 2007

Symposium on children born with clubfoot

We often think that high-tech, surgical methods are the best approach when it comes to medical care. But for children born with clubfoot, there is growing, worldwide interest in a low-tech, non-surgical method of treatment that is more effective, less expensive and easier to provide than surgery.

3 Sep 2007

FDA approves Somatuline Depot to treat acromegaly

The U.S. Food and Drug Administration has approved Somatuline Depot (lanreotide acetate injection) for the treatment of acromegaly, a rare and potentially life threatening disease in adults caused by abnormal secretion of growth hormone (GH), commonly from a benign tumor located in the pituitary gland located in the brain.

30 Aug 2007

FDA grants orphan drug designation to TREANDA

Cephalon, Inc. announced today that the U.S. Food and Drug Administration (FDA) Office of Orphan Products Development granted orphan drug designation for the company's investigational therapy, TREANDA (bendamustine HCl), for the treatment of chronic lymphocytic leukemia (CLL).

29 Aug 2007

New Cockayne syndrome discovery

Scientists of the German Cancer Research Center (Deutsches Krebsforschungszentrum, DKFZ) have shown that the genetic defect that causes Cockayne Syndrome affects a key function of the cell – the transcription of genes coding for ribosomal RNA.

21 Aug 2007

Pharmion and MethylGene announce orphan drug designation for treatment of Hodgkin's lymphoma

Pharmion Corporation, along with its collaborator MethylGene Inc., today announced that the U.S. Food and Drug Administration (FDA) has designated the Companies' histone deacetylase (HDAC) inhibitor, MGCD0103, as an Orphan Drug for the treatment of Hodgkin's lymphoma in the United States.

21 Aug 2007

Vaccines approved by the FDA are at least a million times safer than the viruses that they prevent

For more than 200 years, vaccines have played an important role in the prevention of infectious diseases.

2 Aug 2007

Gluten-free foods - growing market - taste issues

An estimated 2 million Americans are afflicted with celiac disease, an intolerance for food containing wheat, and the market for gluten-free products is booming even while food companies and researchers have yet to fully solve their greatest challenge - making products taste good.

31 Jul 2007

New understanding of Sly Syndrome

The scientist who discovered “Sly Syndrome” nearly four decades ago and a team of colleagues at Saint Louis University are a step closer to finding an approach to treat the rare genetic disease.

26 Jul 2007

Higher child leukaemia rates near nuclear facilities

Leukaemia rates in children and young people are elevated near nuclear facilities, but no clear explanation exists to explain the rise, according to a research review published in the July issue of European Journal of Cancer Care.

20 Jul 2007

Higher leukaemia rates in children living near nuclear facilities

19 Jul 2007

Discovery of enzyme critical in DNA replication

In this week's issue of Science, researchers from the National Institute of Environmental Health Sciences (NIEHS) and Umea University in Sweden report an important discovery about a critical new role that an enzyme called DNA polymerase epsilon plays in replicating DNA in higher organisms such as yeast and perhaps even humans.

9 Jul 2007

Goltz syndrome gene found

Researchers funded in part by the National Institutes of Health have identified the gene that accounts for most cases of Goltz syndrome, a rare skin disorder that can also affect bone and eye development.

23 Jun 2007

breakthrough in the treatment of idiopathic pulmonary haemosiderosis

Researchers at Queen Mary University London and the University of Leicester and have announced a potential breakthrough in the treatment of a rare but devastating medical condition that can affect children and young people.

5 Jun 2007

Breakthrough in idiopathic pulmonary haemosiderosis

3 Jun 2007

New discoveries on genes implicated in Fanconi anaemia

An international research consortium, which included the participation of the Mutagnesi Group from the UAB, has made outstanding progress in the study of Fanconi anaemia.

3 Jun 2007

New biotech company develops UD-patented technologies for repairing genes

OrphageniX Inc., a new biotechnology company founded by University of Delaware researchers, has been established in Wilmington to develop and commercialize UD-patented technologies for repairing genes that cause rare, hereditary diseases such as sickle cell anemia and spinal muscular atrophy.

25 Apr 2007

Johns Hopkins develops pancreas cancer risk software tool

People with a family history of pancreas cancer now have a way to accurately predict their chance of carrying a gene for hereditary pancreas cancer and their lifetime risk of developing the disease.

17 Apr 2007

FDA approves Ceprotin to treat severe congenital protein C deficiency

The U.S. Food and Drug Administration (FDA) today licensed Ceprotin, the first biologic treatment for patients with a rare genetic defect that can cause a potentially life-threatening clotting disorder.

30 Mar 2007