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ASEBIO to host Biospain 2010

The Spanish Bioindustry Association (ASEBIO), in collaboration with the Navarre Society of Development (SODENA), and the government of Navarre, will host BioSpain 2010 the biennial international gathering of experts in the biotechnology sector, in Pamplona from September 29th to October 1st. BioSpain 2010 is honoured to welcome three lecturers of international prestige: Mr. Carlos Cordon-Cardo, Vice-Chair of Pathology & Professor, Columbia University; Mr. Larry Fritz, President & CEO, Covella Pharmaceuticals Inc. and Life Science Venture Partner, Westfield Capital Management Company LP; and Mr. Conny Bogentoft, CEO Karolinska Development AB.

8 Aug 2010

U.S. Senate passes I-ACT to enable patients with rare disease to participate in clinical trials

The U.S. Senate last night passed the "Improving Access to Clinical Trials Act" (I-ACT), a bipartisan piece of legislation championed by the Cystic Fibrosis Foundation, its advocates and 120 other health advocacy organizations.The legislation enables patients with rare diseases to participate in clinical trials without losing eligibility for public healthcare benefits.

7 Aug 2010

Kids v Cancer applauds Senators for Creating Hope Act of 2010

Kids v Cancer applauds Senators Sam Brownback (R-KS), Sherrod Brown (D-OH), and Al Franken (D-MN) for their dedication to curing childhood diseases and supporting the bipartisan "Creating Hope Act of 2010."

7 Aug 2010

PFF announces introduction of Pulmonary Fibrosis Research Enhancement Act in US Senate

The Pulmonary Fibrosis Foundation (PFF) today announced that the Pulmonary Fibrosis Research Enhancement Act (PFREA) was introduced in the United States Senate. The PFREA was introduced by Senators Patty Murray (D-WA) and Mike Crapo (R-ID), and the PFF has offered its unconditional endorsement to pass this important legislation on behalf of the pulmonary fibrosis community.

5 Aug 2010

Carfilzomib could become new option for patients with relapsed myeloma, IMF says

The International Myeloma Foundation, the oldest and largest foundation dedicated to improving the life and care of myeloma patients, today said promising data suggest a new drug called carfilzomib could become an important new option for patients whose myeloma stops responding to other therapies.

4 Aug 2010

Acorda second-quarter net loss decreases to $6.8 million

Acorda Therapeutics, Inc. today announced its financial results for the second quarter of 2010.

3 Aug 2010

Octapharma USA receives confirmation of orphan drug exclusivity from FDA for wilate

Octapharma USA has received confirmation of orphan drug exclusivity from the U.S. Food and Drug Administration for wilate (von Willebrand Factor/Factor VIII Concentrate, Human), the replacement therapy developed specifically for von Willebrand Disease. The approval comes from the FDA Office of Orphan Products Development, which helps to advance the development of products that demonstrate promise for the treatment of rare diseases.

2 Aug 2010

Taligen Therapeutics to highlight three pipeline candidates at XXIII ICW

Taligen Therapeutics, a biotechnology company developing therapies that regulate the complement system to treat inflammatory and immune diseases, today announced that preclinical data on three of its pipeline candidates will be highlighted at the XXIII International Complement Workshop taking place August 1-5, 2010, in New York City.

2 Aug 2010

Researchers identify genetic mutation for Uner Tan Syndrome

A team of researchers from leading universities in Turkey and the United States have reported that they have identified the genetic mutation responsible for the Uner Tan Syndrome (UTS) using advanced genetic capture and sequencing technologies from Roche. The combined genomic technologies enabled the team of scientists to identify the causative mutation of this debilitating disease after years of mystery.

30 Jul 2010

Researchers discover enzyme crucial for DNA repair

Researchers have discovered an enzyme crucial to a type of DNA repair that also causes resistance to a class of cancer drugs most commonly used against ovarian cancer.Scientists from The University of Texas MD Anderson Cancer Center and the Life Sciences Institute of Zhejiang University in China report the discovery of the enzyme and its role in repairing DNA damage called cross-linking in the Science Express advance online publication of Science.

30 Jul 2010

Isis Pharmaceuticals receives $5M payment from GSK for dug development

Isis Pharmaceuticals, Inc. announced today that it has earned a $5 million milestone payment from GlaxoSmithKline related to the designation as a development candidate of a drug to treat an undisclosed rare and serious disease. Isis will develop the drug to Phase 2 proof-of-concept, at which time GSK has the option to license it.

29 Jul 2010

First patient enrolled in Phase 3 SyNAPSe study of BHR-100 for TBI

BHR Pharma, LLC announced today that the first patient has been enrolled in its SyNAPSe study, a global, Phase 3, pivotal trial to evaluate the safety and effectiveness of its intravenous progesterone infusion product (BHR-100) as a neuroprotective agent for treating severe (Glasgow Coma Scale scores of 4-8) traumatic brain injury patients.

23 Jul 2010

Prevalence of HD substantially underestimated in the UK

The prevalence of Huntington's disease is substantially underestimated in the UK, with significant implications for those affected, the healthcare system, and research. New estimates of prevalence, and their implications, are discussed in a comment published in an upcoming Lancet, written by Professor Sir Michael Rawlins, who is the Chairman of the UK National Institute for Health and Clinical Excellence, but writes in his capacity as an Honorary Professor of the London School of Hygiene and Tropical Medicine, UK.

23 Jul 2010

Burden of funding, driving research on rare diseases too often falls upon patients, families: NORD

An advocate for people with rare diseases today told a U.S. Senate committee that the burden of funding and driving research on rare diseases too often falls upon patients and their families.

22 Jul 2010

Fera acquires investigational new drug for treatment of fungal otitis externa

Fera Pharmaceuticals announces the acquisition of an investigational new drug to treat fungal otitis externa (FOE).

22 Jul 2010

Scientists comment on two clinical trials that use Rituximab to treat small-vessel vasculitis

Two UNC School of Medicine faculty authored an editorial in the July 15, 2010 New England Journal of Medicine commenting on results from two clinical trials using the drug Rituximab to treat small-vessel vasculitis, a rare autoimmune disease.

15 Jul 2010

New research focuses on calcium channels in lysosome membrane

In cells, as in cities, disposing of garbage and recycling anything that can be reused is an essential service. In both city and cell, health problems can arise when the process breaks down.

15 Jul 2010

KRX-0401 receives Orphan Drug designation from FDA for treatment of neuroblastoma

Keryx Biopharmaceuticals, Inc. today announced that KRX-0401 (perifosine) has received Orphan Drug designation from the U.S. Food and Drug Administration for the treatment of neuroblastoma, a cancer of the nervous system affecting mostly children and infants for which there are no FDA approved therapies.

15 Jul 2010

Aeterna Zentaris' partner Keryx receives FDA orphan-drug designation for perifosine in neuroblastoma

Aeterna Zentaris Inc., a late-stage drug development company specialized in oncology and endocrine therapy, today announced that its partner, Keryx Biopharmaceuticals, Inc., has been granted orphan-drug designation by the U.S. Food and Drug Administration for perifosine, Aeterna Zentaris' novel, potentially first-in-class, oral Akt inhibitor, for the treatment of neuroblastoma.

15 Jul 2010

Biogen Idec, Swedish Orphan Biovitrum present results from rFIXFc Phase 1/2a study in hemophilia B

Biogen Idec and Swedish Orphan Biovitrum AB today announced results from a Phase 1/2a open-label, dose-escalation, safety and pharmacokinetic study of the companies' long-lasting, fully-recombinant factor IX Fc fusion protein (rFIXFc) in hemophilia B patients. The data, which were presented at the World Federation of Hemophilia Congress in Buenos Aires, Argentina, on July 11, 2010, showed that rFIXFc was well tolerated and demonstrated an approximately three-fold increase in half-life compared to historical data for existing therapies.

12 Jul 2010

Rare Disease News and Research

ASEBIO to host Biospain 2010

U.S. Senate passes I-ACT to enable patients with rare disease to participate in clinical trials

Kids v Cancer applauds Senators for Creating Hope Act of 2010

PFF announces introduction of Pulmonary Fibrosis Research Enhancement Act in US Senate

Carfilzomib could become new option for patients with relapsed myeloma, IMF says

Acorda second-quarter net loss decreases to $6.8 million

Octapharma USA receives confirmation of orphan drug exclusivity from FDA for wilate

Taligen Therapeutics to highlight three pipeline candidates at XXIII ICW

Researchers identify genetic mutation for Uner Tan Syndrome

Researchers discover enzyme crucial for DNA repair

Isis Pharmaceuticals receives $5M payment from GSK for dug development

First patient enrolled in Phase 3 SyNAPSe study of BHR-100 for TBI

Prevalence of HD substantially underestimated in the UK

Burden of funding, driving research on rare diseases too often falls upon patients, families: NORD

Fera acquires investigational new drug for treatment of fungal otitis externa

Scientists comment on two clinical trials that use Rituximab to treat small-vessel vasculitis

New research focuses on calcium channels in lysosome membrane

KRX-0401 receives Orphan Drug designation from FDA for treatment of neuroblastoma

Aeterna Zentaris' partner Keryx receives FDA orphan-drug designation for perifosine in neuroblastoma

Biogen Idec, Swedish Orphan Biovitrum present results from rFIXFc Phase 1/2a study in hemophilia B

Designing fluidic architecture for scalable diagnostic platforms

Evaluating new approaches for rare disease genomics

Expanding access to NMR with the Fourier 80 Duo

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