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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

Cequent Pharmaceuticals proposes the first test of an orally administered RNA interference drug in humans

Cequent Pharmaceuticals, a pioneer in the development of novel products to deliver RNAi-based treatments to prevent and treat human disease, has announced that it has filed its first IND (investigational new drug) application with the U.S. Food and Drug Administration (FDA).

12 Nov 2009

NMO patients are misdiagnosed with Multiple Sclerosis, says Mayo Clinic neurologist

Thousands of Neuromyelitis Optica (NMO) patients are potentially being misdiagnosed with Multiple Sclerosis (MS), according to Mayo Clinic Neurologist Sean Pittock, M.D., largely due to lack of awareness of NMO within the medical community.

12 Nov 2009

ResearchMatch: A free meeting place for researchers

A new national initiative involving UT Southwestern Medical Center will match volunteers who want to take part in medical research studies with the scientists who are leading those studies.

12 Nov 2009

Enzon Announces to sell its specialty pharmaceutical business to the sigma-tau Group for $300 million

Enzon Pharmaceuticals, Inc. (Nasdaq: ENZN) today announced that it has entered into a definitive agreement to sell its specialty pharmaceutical business to the sigma-tau Group for $300 million plus an additional amount of up to $27 million based on success milestones. Enzon will also receive royalties of 5 to 10 percent on incremental net sales above a 2009 baseline amount from Enzon’s four marketed specialty pharmaceutical products through 2014.

9 Nov 2009

PPMD awards $75,000 grant to University of California professor for Duchenne muscular dystrophy research

Patricia A. Furlong, Founding President and CEO of Parent Project Muscular Dystrophy (PPMD), the largest non-profit organization in the United States focused on finding a cure for Duchenne muscular dystrophy (Duchenne), announced today the latest recipient of the End Duchenne Grant Award Program.

6 Nov 2009

FDA grants orphan drug designation for Sunesis Pharmaceuticals' voreloxin

Sunesis Pharmaceuticals, Inc. today announced that the U.S. Food and Drug Administration has granted voreloxin orphan drug designation for the treatment of acute myeloid leukemia (AML). Sunesis is currently conducting two Phase 2 clinical trials of voreloxin in AML: a single-agent study, known as REVEAL-1, of voreloxin in newly diagnosed elderly AML patients unlikely to benefit from standard induction chemotherapy and a study evaluating voreloxin in combination with cytarabine in relapsed/refractory AML.

6 Nov 2009

Canadian patients have less access to the latest medicines, says report

Canadian patients have less access to the latest medicines when compared to the world's developed countries, concludes a comprehensive new report released today. The Report shows Canada ranks 20th out of 25 OECD countries in public reimbursement of new drug therapies. Canada's grade of 56% is comparable with Iceland, Slovakia and Turkey.

4 Nov 2009

Shire announces third quarter 2009 financial results

Shire plc, the global specialty biopharmaceutical company, announces results for the three months to September 30, 2009.

31 Oct 2009

Child Neurology Foundation raises $30,000 during Infantile Spasms Awareness Week

The Child Neurology Foundation (CNF) today announced that $30,000 was raised during Infantile Spasms Awareness Week (October 11-17) that will go towards researching the cause and treatment of this ultra-rare form of epilepsy.

28 Oct 2009

BioMarin Pharmaceutical acquires Huxley Pharmaceuticals

Rodman & Renshaw Capital Group, Inc. announced today that its affiliate, Aceras BioMedical LLC (Aceras), has sold Huxley Pharmaceuticals Inc. (Huxley), a portfolio company, to BioMarin Pharmaceutical Inc. (NASDAQ: BMRN). Huxley was established by Aceras in August 2008 with a focus on developing therapeutic products for rare diseases of the nervous system.

27 Oct 2009

Modified NAGA: New enzyme for enzyme replacement therapy

A new study uses a creative structure-based remodeling strategy to design a therapeutic protein that exhibits significant advantages over currently available treatments for a rare disease that often leads to cardiac and renal failure. The research, published by Cell Press on October 22nd in the American Journal of Human Genetics, describes a new and highly promising candidate for enzyme replacement therapy (ERT) for Fabry disease.

23 Oct 2009

Pfizer announces first recipient of its Ripples of Hope Trainee Award in Mental Health

Pfizer Canada is pleased to announce Valeria Uribe as the first recipient of the newly established Pfizer Ripples of Hope Trainee Award in Mental Health. Ms. Uribe is currently working towards a Masters in Science at the University of British Columbia in her pursuit of a career in neuroscience.

21 Oct 2009

NCCN Guidelines for Acute Myeloid Leukemia updated

The National Comprehensive Cancer Network (NCCN) recently made updates to the NCCN Clinical Practice Guidelines in Oncology(TM) for Acute Myeloid Leukemia, a cancer that starts inside the bone marrow and the most common acute leukemia affecting adults.

20 Oct 2009

MDS secures international cooperation to help Canadian teenager with malignant blood disorder

The Myelodysplastic Syndromes (MDS) Foundation says a wide range of international cooperation has been secured to help 18-year-old Wes Laporte of Ontario get treatment for a life-threatening condition in the US.

15 Oct 2009

University of North Carolina awarded grant to continue research on rare disease-causing gene mutations

The University of North Carolina at Chapel Hill has been awarded a five-year, $6.2 million renewal grant to continue its work as part of the National Institutes of Health's Rare Diseases Clinical Research Network (RDCRN).

14 Oct 2009

NIH funds the University of California's consortium for immune disorder research

The University of California, San Francisco has been designated to lead a new consortium that will study a group of severe immune disorders known as primary immunodeficiencies and aims to improve treatment for these often life-threatening diseases. The Primary Immune Deficiency Treatment Consortium comprises 13 centers throughout the United States and has a $6.25 million funding commitment over five years from the National Institutes of Health.

13 Oct 2009

CNF launches new educational website centered on infantile spasms

The Child Neurology Foundation (CNF) today announced the launch of a new educational website centered on infantile spasms (IS). The website, www.infantilespasmsinfo.org, is an educational resource developed by the CNF as one of the key components of Infantile Spasms Awareness Week.

12 Oct 2009

Antidotes effective for treating acute liver failure patients in early stages of disease

The antidote for acute liver failure caused by acetaminophen poisoning also can treat acute liver failure due to most other causes if given before severe injury occurs, UT Southwestern Medical Center researchers and their colleagues at 21 other institutions have found.

8 Oct 2009

Rare Disease News and Research

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

Myeloma UK launches innovative clinical trial network

Cequent Pharmaceuticals proposes the first test of an orally administered RNA interference drug in humans

NMO patients are misdiagnosed with Multiple Sclerosis, says Mayo Clinic neurologist

ResearchMatch: A free meeting place for researchers

Enzon Announces to sell its specialty pharmaceutical business to the sigma-tau Group for $300 million

PPMD awards $75,000 grant to University of California professor for Duchenne muscular dystrophy research

FDA grants orphan drug designation for Sunesis Pharmaceuticals' voreloxin

Canadian patients have less access to the latest medicines, says report

Shire announces third quarter 2009 financial results

Child Neurology Foundation raises $30,000 during Infantile Spasms Awareness Week

BioMarin Pharmaceutical acquires Huxley Pharmaceuticals

Modified NAGA: New enzyme for enzyme replacement therapy

Pfizer announces first recipient of its Ripples of Hope Trainee Award in Mental Health

NCCN Guidelines for Acute Myeloid Leukemia updated

MDS secures international cooperation to help Canadian teenager with malignant blood disorder

University of North Carolina awarded grant to continue research on rare disease-causing gene mutations

NIH funds the University of California's consortium for immune disorder research

CNF launches new educational website centered on infantile spasms

Antidotes effective for treating acute liver failure patients in early stages of disease

Achieving and maintaining ISO/IEC 27001 and BS 10012 certifications

Expanding research and clinical options for children with cancer

Examining how pain could play a direct protective role in the gut

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