Rare Disease News and Research

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Need for education for primary care physicians on rarely seen cancers

Many primary care physicians may lack the necessary knowledge when it comes to recognizing the signs, symptoms and making proper diagnoses in cases of blood cancers such as leukemia and lymphoma, a recent survey indicates.

10 Dec 2007

New method for early diagnosis of pulmonary hypertension

Researchers at the University of Granada ( UGR ) from the Systemic Autoimmune Diseases Unit and the Cardiology Service of the Hospital Clínico San Cecilio in Granada have developed an innovative system which will help doctors make the earliest diagnosis of pulmonary hypertension possible, which is the main cause of death for patients suffering from scleroderma, a rare disease which affects approximately 1,200 out of every million people.

14 Nov 2007

Blood gene expression signatures predict exposure levels

In a new study, researchers found they could detect toxic levels of acetaminophen in laboratory animals by analyzing gene expression in the blood.

1 Nov 2007

NIH and India partner to develop low-cost health-care technologies

The National Institute of Biomedical Imaging and Bioengineering (NIBIB), part of the National Institutes of Health (NIH), and the Department of Biotechnology (DBT) of the Ministry of Science and Technology of the Republic of India, have entered into a bilateral agreement to develop low-cost health-care technologies aimed at the medically underserved.

22 Oct 2007

Myositis featured on Discovery Channel

LaShan Davis, featured this week on The Discovery Channel's “Mystery Diagnosis” spent three 10-hour days filming the segment and reliving the terrifying time she spent waiting for her “mystery disease” to be diagnosed.

17 Oct 2007

Folic acid supplements dramatically lower blood arsenic levels in those exposed to contaminated water

A new study conducted in Bangladesh finds that folic acid supplements can dramatically lower blood arsenic levels in individuals chronically exposed to arsenic-contaminated drinking water.

10 Oct 2007

Paving the way for future pan-European clinical trials

Pan-European collaboration is important for many clinical trials and essential for trials that are investigating treatments for rare diseases.

10 Oct 2007

Completion of first clinical trial of Apatone for cancer treatment

In a significant advancement in the ongoing battle against cancer, a group of researchers from Summa Health System, IC-MedTech and other institutions have completed the first ever FDA-approved human clinical trial of Apatone®.

8 Oct 2007

Discovery of new pathway causing cell death related to frontotemporal dementia and ALS

Scientists have discovered a link between a mutated gene and a protein found in dead brain cells of people who suffer from a form of dementia and other neurological disorders.

27 Sep 2007

Rochester receives $1.2 million to battle rare childhood disease

"For an orphan disease like Batten, which has such a clear-cut course of deterioration and no present cure, this grant is transformative both for these families and for us, as clinicians and researchers."

12 Sep 2007

Mutations in the insulin gene can cause permanent neonatal diabetes

Mutations in the insulin gene can cause permanent neonatal diabetes, an unusual form of diabetes that affects very young children and results in lifelong dependence on insulin injections, report researchers from the University of Chicago and Peninsula University (Exeter, UK) in Sept. 18, 2007, issue of the Proceedings of the National Academy of Sciences, published early online.

11 Sep 2007

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