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Scientists start clinical trial of EDP-788 oral antibiotic to treat MRSA infections

Scientists have begun the first human clinical trial of EDP-788, an investigational oral antibiotic intended to treat methicillin-resistant Staphylococcus aureus (MRSA) infections.

27 Jan 2014

Researchers find novel drug combination approach to speed fight against cancer

Cancer is the leading cause of death in Canada and is responsible for about 30% of all deaths, according to the Canadian Cancer Society. Despite the urgent need for new and effective drugs, it remains extremely difficult to develop anti-cancer therapies in a timely way.

27 Jan 2014

CytRx initiates aldoxorubicin Phase 2 trial in HIV-infected patients with Kaposi's sarcoma

CytRx Corporation, a biopharmaceutical research and development company specializing in oncology, today announced it has initiated a Phase 2 clinical trial to determine preliminary efficacy and safety of aldoxorubicin for HIV-infected patients with Kaposi's sarcoma.

23 Jan 2014

BioLineRx's BL-8040 gets FDA Orphan Drug Designation for stem cell mobilization treatment

BioLineRx, a clinical-stage biopharmaceutical company dedicated to identifying, in-licensing and developing promising therapeutic candidates, announced today that it has received notice from the U.S. Food & Drug Administration confirming an Orphan Drug Designation of BL-8040 as a treatment for stem cell mobilization, in addition to the Orphan Drug Designation previously granted to BL-8040 as a treatment for Acute Myeloid Leukemia.

23 Jan 2014

ACMG’s Genomics Annual Clinical Genetics Meeting showcases latest breakthroughs in genetics research

Do you cover genetics, genomics, healthcare or medicine? The media are invited to register now for the American College of Medical Genetics and Genomics Annual Clinical Genetics Meeting, March 25-29, 2014 at the Nashville Convention Center.

23 Jan 2014

Joint research program in SMA enters initial stage of clinical development

PTC Therapeutics, Inc., the SMA Foundation, and Roche, announced today that their joint research program in Spinal Muscular Atrophy has entered the first stage of clinical development aiming to assess safety and tolerability of an oral compound in healthy volunteers.

22 Jan 2014

Alexion's Soliris gets FDA orphan drug designation for DGF prevention in renal transplant patients

Alexion Pharmaceuticals today announced that the U.S. Food and Drug Administration has granted an orphan drug designation to Soliris (eculizumab), a first-in-class terminal complement inhibitor, for the prevention of delayed graft function in renal transplant patients.

21 Jan 2014

New technology efficiently transfers investigational antibodies from blood via blood-brain barrier

Today the scientific journal Neuron published results on the Roche-designed Brain Shuttle technology that efficiently transfers investigational antibodies from the blood through the blood-brain barrier (BBB) into the brain in preclinical models.

10 Jan 2014

Researchers use stem cells to study bone disorders from patients afflicted with genetic bone disease

Researchers have developed a new way to study bone disorders and bone growth, using stem cells from patients afflicted with a rare, genetic bone disease.

9 Jan 2014

Study shows ability to make living human cells from biobanked brain tissue of Alzheimer's patients

Scientists at The New York Stem Cell Foundation (NYSCF) Research Institute, working in collaboration with scientists from Columbia University Medical Center (CUMC), for the first time generated induced pluripotent stem (iPS) cells lines from non-cryoprotected brain tissue of patients with Alzheimer's disease.

7 Jan 2014

FDA accepts NPS Pharmaceuticals' Natpara BLA for review

NPS Pharmaceuticals, Inc., a global biopharmaceutical company pioneering and delivering therapies that transform the lives of patients with rare diseases worldwide, today announced that the U.S. Food and Drug Administration has accepted and filed for review the company's Biologics License Application for Natpara (recombinant human parathyroid hormone 1-84, (rhPTH[1-84])) for the treatment of hypoparathyroidism.

7 Jan 2014

FDA OOPD grants orphan drug designation to Alitair's Erdosteine for treatment of bronchiectasis

Alitair Pharmaceuticals, Inc., today announced that the Office of Orphan Products Development of the Food and Drug Administration has granted orphan drug designation for its drug candidate, Erdosteine, for the treatment of bronchiectasis. Erdosteine is a mucolytic with antibacterial, anti-inflammatory, and antioxidant properties.

7 Jan 2014

NPS Pharmaceuticals' Natpara gets orphan drug designation for treatment of hypoparathyroidism

NPS Pharmaceuticals, Inc., a global biopharmaceutical company pioneering and delivering therapies that transform the lives of patients with rare diseases, today announced that the European Commission has granted orphan drug designation to Natpara (recombinant human parathyroid hormone (rhPTH[1-84]) for the treatment of hypoparathyroidism.

3 Jan 2014

NPSP to conduct open-label study to evaluate teduglutide in pediatric patients with SBS

NPS Pharmaceuticals, Inc. (NASDAQ: NPSP), a global biopharmaceutical company pioneering and delivering therapies that transform the lives of patients with rare diseases worldwide, today announced that the company has initiated a global registration study of teduglutide in pediatric patients with Short Bowel Syndrome (SBS) who are dependent on parenteral support.

3 Jan 2014

Diflunisal drug reduces neurological decline in patients with familial transthyretin amyloidosis

A recent study led by researchers from the Amyloidosis Center at Boston University School of Medicine and Boston Medical Center demonstrates that diflunisal, a generic anti-inflammatory drug, successfully reduced neurological decline and preserved the quality of life in patients with familial transthyretin amyloidosis. Diflunisal is an inexpensive and safe medication marketed over the past 40 years for arthritis and pain.

25 Dec 2013

U.S. FDA grants Veloxis Orphan Drug status for Envarsus

Veloxis Pharmaceuticals A/S, today announced that Envarsus was granted Orphan Drug status by the U.S. Food and Drug Administration (FDA) for prophylaxis of organ rejection in patients receiving allogenic kidney transplants.

24 Dec 2013

Six common diseases arise from DNA changes, finds new research

Many rare disorders are caused by gene mutation, like sickle cell anemia. Yet until now the underlying genetic cause of more common conditions - for example, rheumatoid arthritis - has evaded scientists for years.

22 Dec 2013

ORIX USA Health partners with TGen and TD2 to support Arizona's biomedical economy

ORIX USA Health and Life Sciences has completed an investment into Translational Drug Development (TD2) through the purchase of equity from the Translational Genomics Research Institute (TGen).

20 Dec 2013

Researchers treat osteogeneis imperfecta babies in utero by injecting bone-forming stem cells

Osteogeneis imperfecta (OI) is a congenital bone disease that causes stunted growth and repeated, painful fracturing. Ultrasound scans can reveal fractures already in the fetus, and now an international team of researchers from Sweden, Singapore and Taiwan have treated two babies in utero by injecting bone-forming stem cells.

17 Dec 2013

Johns Hopkins researchers use lung cancer drug to shrink chordoma in mice

Johns Hopkins researchers say that a drug approved to treat lung cancer substantially shrank tumors in mice that were caused by a rare form of bone cancer called chordoma.

12 Dec 2013

Rare Disease News and Research

Scientists start clinical trial of EDP-788 oral antibiotic to treat MRSA infections

Researchers find novel drug combination approach to speed fight against cancer

CytRx initiates aldoxorubicin Phase 2 trial in HIV-infected patients with Kaposi's sarcoma

BioLineRx's BL-8040 gets FDA Orphan Drug Designation for stem cell mobilization treatment

ACMG’s Genomics Annual Clinical Genetics Meeting showcases latest breakthroughs in genetics research

Joint research program in SMA enters initial stage of clinical development

Alexion's Soliris gets FDA orphan drug designation for DGF prevention in renal transplant patients

New technology efficiently transfers investigational antibodies from blood via blood-brain barrier

Researchers use stem cells to study bone disorders from patients afflicted with genetic bone disease

Study shows ability to make living human cells from biobanked brain tissue of Alzheimer's patients

FDA accepts NPS Pharmaceuticals' Natpara BLA for review

FDA OOPD grants orphan drug designation to Alitair's Erdosteine for treatment of bronchiectasis

NPS Pharmaceuticals' Natpara gets orphan drug designation for treatment of hypoparathyroidism

NPSP to conduct open-label study to evaluate teduglutide in pediatric patients with SBS

Diflunisal drug reduces neurological decline in patients with familial transthyretin amyloidosis

U.S. FDA grants Veloxis Orphan Drug status for Envarsus

Six common diseases arise from DNA changes, finds new research

ORIX USA Health partners with TGen and TD2 to support Arizona's biomedical economy

Researchers treat osteogeneis imperfecta babies in utero by injecting bone-forming stem cells

Johns Hopkins researchers use lung cancer drug to shrink chordoma in mice

Sensing a Healthier Future

Achieving and maintaining ISO/IEC 27001 and BS 10012 certifications

Expanding research and clinical options for children with cancer

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