Rare Disease News and Research

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New drug use screening tools for physicians

The National Institute on Drug Abuse (NIDA), part of the National Institutes of Health, today unveiled its first comprehensive Physicians' Outreach Initiative, NIDAMED, which gives medical professionals tools and resources to screen their patients for tobacco, alcohol, illicit, and nonmedical prescription drug use.

20 Apr 2009

New AIDS research training grants awarded for projects in 15 countries

The Fogarty International Center of the National Institutes of Health has awarded seven grants totaling almost $2.7 million to train HIV/AIDS researchers in 15 low- and middle-income countries.

14 Apr 2009

Tolera Therapeutics granted orphan drug status for organ transplant rejection lead candidate drug

Tolera Therapeutics, Inc., was granted orphan drug status by the United States Food and Drug Administration (FDA) for its initial lead candidate, TOL101, for prophylaxis of acute rejection of solid organ transplantation.

12 Apr 2009

Better health and prosperity in Europe via improved clinical research

Improved patient-oriented research in Europe will benefit European citizens and the European medical industry and facilitate the transfer of scientific discoveries from the laboratory bench to the bedside. For Europe and for the rest of the world this effort will be of great importance for the quality of life of individuals and the wellbeing of society as a whole.

12 Mar 2009

SOX9 protein may be a potential target for melanoma treatment

In a new study released today, researchers have shown that a specific protein plays an important role in inhibiting the development and spread of melanoma tumors in mouse and in human skin models.

11 Mar 2009

Immune-based therapy approved in Europe for pediatric cancer patients

The European Commission, which oversees legislation and regulation for the European Union, has approved a therapy for pediatric patients with non-metastatic, resectable osteosarcoma, a type of bone cancer.

11 Mar 2009

Genetic mutations suggest link between type 1 diabetes and common viral infection

Scientists from Cambridge University have discovered four rare mutations of a gene associated with type 1 diabetes (T1D) that reduce the risk of developing the disease.

5 Mar 2009

Launch of new foundation for Batten disease

Leading scientists, medical researchers and affected families joined together today to launch Beyond Batten Disease Foundation, an organization committed to finding a cure for this rare disease that claims the lives of children.

4 Mar 2009

Molecule provides clues about how HPV may lead to cancer

New research shows for the first time that certain types of human papillomavirus (HPV), which cause cervical and some other types of cancer, can inhibit the production of a tiny single-stranded RNA called microRNA 34a, or miR-34a.

4 Mar 2009

Scientists identify lab-made proteins that neutralize a broad range of influenza A viruses

Scientists have identified a small family of lab-made proteins that neutralize a broad range of influenza A viruses, including the H5N1 avian virus, the 1918 pandemic influenza virus and seasonal H1N1 flu viruses.

23 Feb 2009

FDA approves orphan drug ATryn anticoagulant

The U.S. Food and Drug Administration has issued its first approval for a biological product produced by genetically engineered (GE) animals.

8 Feb 2009

Study tests salsalate anti-inflammatory for poorly controlled type 2 diabetes

Researchers in 20 medical centers across the country are enrolling adults with type 2 diabetes who have poorly controlled blood glucose to participate in a clinical study, Targeting Inflammation with Salsalate in Type 2 Diabetes (TINSAL-T2D).

5 Feb 2009

Discovery of new cause of ozone wheezing and potential treatments

Researchers at the National Institute of Environmental Health Sciences (NIEHS), part of the National Institutes of Health, and Duke University have discovered a cause of airway irritation and wheezing after exposure to ozone, a common urban air pollutant.

3 Feb 2009

Brain cells called astrocytes, not neurons, lull the brain to sleep

Brain cells called astrocytes help to cause the urge to sleep that comes with prolonged wakefulness, according to a study in mice, funded by the National Institutes of Health.

2 Feb 2009

FDA approves RiaSTAP for bleeding in patients with congenital fibrinogen deficiency

The U.S. Food and Drug Administration has licensed RiaSTAP, an orphan drug for the treatment of bleeding in patients with a rare genetic defect known as congenital fibrinogen deficiency. Without treatment, these patients are at risk of potentially life-threatening bleeding.

18 Jan 2009