Rare Disease News and Research

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CSL Behring donates protein therapies to World Federation of Hemophilia

In recognition of Rare Disease Day and as part of its ongoing commitment to the global bleeding disorders community, CSL Behring announced today that it is donating 2 million international units (IUs) of protein therapies to the World Federation of Hemophilia.

28 Feb 2015

Rare Disease Day 2015 raises awareness of challenges faced by rare cancer patients

Within the "umbrella" of rare diseases, rare cancer patients confront particular problems. Most rare diseases have an identified genetic origin. In contrast, rare cancers are mainly acquired diseases.

28 Feb 2015

Cipher acquires worldwide rights to three products from Astion

Cipher Pharmaceuticals Inc. today announced it has strengthened its product pipeline by acquiring the worldwide rights to three products from Astion Pharma, a Denmark-based specialty pharmaceutical company.

26 Feb 2015

Tolero's alvocidib receives EMA orphan drug designation for treatment of AML patients

Tolero Pharmaceuticals, Inc., a clinical-stage company developing treatments for serious hematological diseases, today announced that the European Medicines Agency has granted orphan drug designation for alvocidib for the treatment of patients with acute myeloid leukemia (AML).

26 Feb 2015

Isis Pharmaceuticals announces positive results from Phase 1 study of ISIS-PKK Rx for HAE treatment

Isis Pharmaceuticals, Inc. announced today positive results from a Phase 1 study with ISIS-PKKRx. In this study, healthy volunteers treated with ISIS-PKKRx achieved dose-dependent reductions of up to 95 percent in prekallikrein, or PKK. ISIS-PKKRx is a RNA-targeted antisense drug designed to inhibit the production of PKK for the prophylactic treatment of hereditary angioedema (HAE).

24 Feb 2015

Shire announces acquisition of Meritage Pharma

Shire plc and Meritage Pharma, Inc. announced today that Shire has acquired Meritage, a privately-held company, for an upfront fee of $70 million and additional contingent payments based on the achievement of development and regulatory milestones.

24 Feb 2015

Shire announces completion of NPS Pharma acquisition

Shire plc announces the successful completion of the tender offer for all of the outstanding shares of NPS Pharmaceuticals, Inc. and the subsequent acquisition of NPS Pharma.

21 Feb 2015

EC grants orphan drug designation to aTyr Pharma's Resolaris for treatment of FSHD

aTyr Pharma, Inc., a biotherapeutics company engaged in the discovery and development of Physiocrine-based therapeutics to address rare diseases, announced today the European Commission (EC) has granted orphan drug designation to Resolaris for the treatment of facioscapulohumeral muscular dystrophy (FSHD).

19 Feb 2015

Researchers show importance of DNA damage in fine tuning of innate immune system

For the first time scientists from Umeå University show the importance of DNA damage in fine tuning of our innate immune system and hence the ability to mount the optimal inflammatory response to infections and other biological dangers.

18 Feb 2015

Oncolytics Biotech’s REOLYSIN receives Orphan Drug Designation for treatment of pancreatic cancer

Oncolytics Biotech® Inc. ("Oncolytics") (TSX:ONC, NASDAQ: ONCY), a clinical-stage biotechnology company focused on the development of oncolytic viruses as potential cancer therapeutics, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for its lead product candidate, REOLYSIN®, for the treatment of pancreatic cancer.

17 Feb 2015

R. Bryan Miller Symposium to spotlight special focus on rare disease research

The University of California, Davis Department of Chemistry will spotlight a special focus on rare disease research with this year's 15th annual R. Bryan Miller Symposium. Experts in rare diseases will gather at the UC Davis Conference Center March 5 for a conference highlighting the opportunities and challenges in applying cutting edge technologies and "precision medicine" to better treat conditions that together affect millions of people, especially children.

16 Feb 2015

A*STAR's GIS scientists develop new system that can predict treatment targets for cancer

In recent months, several national initiatives for personalized medicine have been announced, including the recently launched precision medicine initiative in the US, driven by rapid advances in genomic technologies and with the promise of cheaper and better healthcare. Significant challenges remain, however, in the management and analysis of genetic information and their integration with patient data.

15 Feb 2015

NanoSmart's ANA-conjugated liposomal doxorubicin receives FDA Orphan Drug Designation

NanoSmart Pharmaceuticals, Inc., a private pharmaceutical company developing nanoparticle drug delivery platforms, has received Orphan Drug Designation from the Food and Drug Administration for one of its lead candidates, antinuclear antibody (ANA) conjugated liposomal doxorubicin.

11 Feb 2015

TAU researchers identify novel genetic mutation as source of specific rare disease

Rare diseases -- those that affect fewer than one in 200,000 people -- are often identified early in life. Some 30 percent of children afflicted by these "orphan diseases" do not live to see their fifth birthday. While the U.S. Orphan Drug Act of 1983 was written into law to promote research on the topic, the cost of identifying the source and progression of these diseases remains prohibitive for many families.

10 Feb 2015

Oncolytics Biotech submits Orphan Drug Designation for high grade gliomas in pediatric patients

Oncolytics Biotech Inc. ("Oncolytics") (TSX:ONC, NASDAQ: ONCY) today announced that it has submitted an application for Orphan Drug Designation to the U.S. Food and Drug Administration ("FDA") for REOLYSIN® for the treatment of high grade gliomas (HGG) in pediatric patients.

9 Feb 2015

Researchers study epidemiology of Ebola Virus Disease to prevent future disease outbreaks

Now, researchers from Arizona State University and Georgia State University are trying to better understand the epidemiology and control of Ebola Virus Disease in order to alleviate suffering and prevent future disease outbreaks from reaching the catastrophic proportions of the current crisis.

9 Feb 2015

Mesothelioma Victims Center implores US homeowners to understand the potential hazards of asbestos

The Mesothelioma Victims Center is urging all US homeowners to familiarize themselves with the dangers of exposure to asbestos in any home constructed with the material as there could be a remote possibility of developing a malignant form of cancer called mesothelioma.

2 Feb 2015

Education can help people better understand individuals with facial paralysis

A little bit of sensitivity training can help people form better first impressions of those with facial paralysis, reducing prejudices against people with a visible but often unrecognizable disability, new research from Oregon State University indicates.

21 Jan 2015

Researchers reveal key factor in understanding elevated cancer risk linked to gene therapy

National Institutes of Health researchers have uncovered a key factor in understanding the elevated cancer risk associated with gene therapy. They conducted research on mice with a rare disease similar to one in humans, hoping their findings may eventually help improve gene therapy for humans. Researchers at the National Human Genome Research Institute, part of NIH, published their research in the Jan. 20, 2015, online issue of the Journal of Clinical Investigation.