Rare Disease News and Research

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A novel approach for funding children's genetic disease research

Parent Project Muscular Dystrophy together with Trisomy 18 Foundation are among 100 charities that earned a $25,000 grant during Round 1 of the Chase Community Giving Challenge on Facebook.

19 Jan 2010

GAO: Some meds had 'extraordinary' price hikes over past eight years

A report from the Government Accountability Office (GAO) issued Monday finds that "[p]rices on a growing number of prescription medications have ballooned in recent years as consolidation in the drug industry leaves fewer companies manufacturing niche medications," The Associated Press reports.

12 Jan 2010

Opinions: U.S. medical emergency response; micronutrients; rotavirus vaccine; antibiotic development

"[D]espite the tireless efforts of public health and health-care workers, America's experience with H1N1 shows that the nation is not prepared to deal with a flu pandemic," former Democratic Senator Bob Graham and former Republican Senator Jim Talent write in Washington Post opinion piece that examines U.S. preparedness for medical emergencies. The writers chaired the Commission on the Prevention of Weapons of Mass Destruction Proliferation and Terrorism.

6 Jan 2010

Senate health care bill will increase patient access and help create high-wage, high-value jobs

“These provisions mean increased access for the patients of today and renewed hope for the patients of tomorrow. In addition, they will help create high-wage, high-value jobs in our nation’s burgeoning biotech sector and help maintain U.S. global leadership in innovation.”

28 Dec 2009

FDA provides guidance on applying for orphan drug designation

As part of its continuing effort to make the agency more transparent and accessible, the FDA has scheduled a series of workshops about orphan drug designation for academics, biotechnology companies, and those unfamiliar with the process. The

24 Dec 2009

University of Florida obtains stimulus grant to establish ataxia consortium

University of Florida neurologists are trying to change that with the help of a $1 million Challenge grant from the National Institute of Neurological Disorders and Stroke to establish a nationwide network of physician-scientists with expertise in clinical ataxia research.

16 Dec 2009

Manchester Pharmaceuticals' Chenodal now available for patients suffering from gallstones

Manchester Pharmaceuticals, Inc.(TM) announced today the availability of Chenodal(TM) 250 mg (chenodiol tablets). On October 22, 2009, the US Food and Drug Administration (FDA) approved Chenodal, an important therapy for patients suffering from gallstones in whom surgery poses an unacceptable health risk due to disease or advanced age.

15 Dec 2009

EC grants orphan drug designation for MP4CO for treating sickle cell disease

Sangart, Inc., a global biopharmaceutical company dedicated to developing life-saving medicines specifically designed to enhance the perfusion and oxygenation of ischemic (oxygen deprived) tissues through targeted oxygen and other gas delivery, today announced that the European Commission (EC) has granted orphan medicinal product designation for MP4CO for the treatment of sickle cell disease.

10 Dec 2009

FDA launches workshop to simplify orphan drug development

The U.S. Food and Drug Administration (FDA) has announced the launch of an orphan drug workshop series--an opportunity for biotechnology companies, pharmaceutical firms, academics and other interested parties to spend two days creating applications for orphan status designation, under the guidance of staff from FDA's Office of Orphan Products Development (OOPD).

9 Dec 2009

BHR Pharma files an IND for its BHR-100 intravenous progesterone infusion product

BHR Pharma, LLC (BHR) announced today that it has filed an Investigational New Drug (IND) application with the U.S. Food and Drug Administration (FDA) for its proprietary BHR-100 intravenous progesterone infusion product.

8 Dec 2009

Stem cell treatment: A new hope for Multiple Sclerosis

RNL Bio Co., Ltd, (www.rnl.co.kr) a leading biopharmaceutical company specialized in adult stem cell therapeutics announced today that a 46-year-old female, Kang Sook Park's Multiple Sclerosis improved tremendously after receiving stem cell treatment. Park was suffering from MS (Multiple Sclerosis), an autoimmune disease that affects the central nervous system (brain and spinal cord).

6 Dec 2009

Allos Therapeutics to launch the COMPLETE registry for patients with PTCL

Allos Therapeutics, Inc. today announced the launch of an international registry designed to address the urgent need to better understand treatment patterns and outcomes for patients with peripheral T-cell lymphoma (PTCL). The registry, known as COMPLETE (Comprehensive Oncology Measures for Peripheral T-cell Lymphoma Treatment), is a global observational study that will enroll patients with newly-diagnosed PTCL and obtain data regarding longitudinal treatment patterns and outcomes.

3 Dec 2009

Pfizer, Protalix team up to develop and market plant-based enzyme for Gaucher’s disease treatment

Pfizer and Protalix (NYSE-Amex: PLX) today announced that they have entered into an agreement to develop and commercialize taliglucerase alfa, a plant-cell expressed form of glucocerebrosidase (GCD) in development for the potential treatment of Gaucher’s disease.

1 Dec 2009

Healthy dietary habits are important for GCA prevention

The gastric cardiac adenocarcinoma (GCA) is a very rare disease. In recent decades, however, the incidence of the GCA has increased dramatically in many Western countries. An increasing trend in GCA is also observed in municipal regions but not in rural regions of China. Another striking feature is the strong male predominance among patients with GCA. Some of the reasons above are still unclear and require further epidemiological investigations.

26 Nov 2009

Roche, Spanish Department of Health to collaborate in Medical Genome Project

Roche and the Department of Health of Junta de Andalucía, Spain have signed an agreement for a cooperation in the Medical Genome Project (MGP), a research project created to analyse the genomes of people with rare diseases and identify the defective gene or genes causing the disease and its behaviour.

From Roche Sequencing and Life Science 24 Nov 2009

Phase IIb/III clinical trial results of CINQUIL for pediatric EoE announced

Ception Therapeutics, Inc., a privately held biopharmaceutical company, and Cephalon, Inc. today announced results from a Phase IIb/III clinical trial for CINQUIL(TM) (reslizumab) as a treatment for pediatric eosinophilic esophagitis (EoE). The study was designed to evaluate improvement in the co-primary endpoints of changes in esophageal eosinophil levels and clinical symptoms.

23 Nov 2009

Scientists use exome sequencing method to discover the cause of mendelian disorder

For the first time, scientists have successfully used a method called exome sequencing to quickly discover a previously unknown gene responsible for a mendelian disorder.

20 Nov 2009

NORD letter conveys support for passage of the Patient Protection and Affordable Care Act

On behalf of the 150 member organizations of the National Organization for Rare Disorders (NORD) representing nearly 30 million patients and families afflicted with one of the 7,000 known rare diseases, we are writing to convey our strong support for passage of the Patient Protection and Affordable Care Act.

20 Nov 2009

Early diagnosis and treatment is crucial for pulmonary hypertension, a rare but deadly disease

We often hear about the perils of high blood pressure, but many Canadians are unaware that high blood pressure in the lungs - known as pulmonary hypertension (PH) - can also have fatal consequences. This month, the Pulmonary Hypertension Association of Canada hopes to raise awareness about PH - a rare but serious disease that can strike at any time and has no regard for race, age or gender.

19 Nov 2009

Myeloma UK launches innovative clinical trial network

Yorkshire myeloma patients are set to be among the first to benefit from an innovative Myeloma Clinical Trial Network launched by Myeloma UK.

16 Nov 2009