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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

CSL Behring LEAD program invites grant requests from organizations assisting people with rare diseases

CSL Behring, a global leader in the plasma-protein biotherapeutics industry, is calling for grant requests through its Local Empowerment for Advocacy Development (LEAD) program. The LEAD program awards funds to United States patient organizations that assist people with rare diseases in advocating for continued access to health care and life-saving plasma protein therapies.

7 Oct 2009

Daily doses of imatinib mesylate double survival rates in Ph+ ALL affected children

Results of a phase two clinical trial published October 5th in the Journal of Clinical Oncology show that adding continuous daily doses of a targeted drug called imatinib mesylate to regular chemotherapy more than doubled three-year survival rates for children with a high risk type of blood cancer called Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL).

7 Oct 2009

Research to explore diagnosis and treatment of rare diseases

The National Institutes of Health announced today a second phase of the Rare Diseases Clinical Research Network (RDCRN) including funds for 19 research consortia.

7 Oct 2009

IDF announces new social networking online website for people with PIDD

People with primary immunodeficiency diseases (PIDD) and their families will now be able to connect with one another and share their experiences through a new multidimensional social networking initiative, the Immune Deficiency Foundation (IDF) announced today. Primary immunodeficiency diseases are a group of rare, chronic illnesses, caused by hereditary or genetic defects in the immune system.

7 Oct 2009

The EURORDIS and NORD rare disease organizations form a partnership

The leading advocacy groups for Americans and Europeans with rare diseases -- the National Organization for Rare Disorders (NORD) and the European Organization for Rare Diseases (EURORDIS) -- have signed a Memorandum of Understanding to join forces on several key strategic initiatives on behalf of rare disease patients and their families.

3 Oct 2009

Post Baccalaureate Premedical Program to begin in January 2010

Keck Graduate Institute (KGI) has established a Post Baccalaureate Premedical Program (PBPM), scheduled to begin in January 2010, for those with an undergraduate degree in science or engineering who are interested in enhancing their skills before applying to medical school.

30 Sep 2009

Combined immunodeficiency results in severe health problems

Researchers at the National Institutes of Health have identified a genetic mutation that accounts for a perplexing condition found in people with an inherited immunodeficiency. The disorder, called combined immunodeficiency, is characterized by a constellation of severe health problems, including persistent bacterial and viral skin infections, severe eczema, acute allergies and asthma, and cancer.

28 Sep 2009

Folotyn approved for the treatment of Peripheral T-Cell Lymphoma

The U.S. Food and Drug Administration has approved Folotyn (pralatrexate), the first treatment for a form of cancer known as Peripheral T-cell Lymphoma (PTCL), an often aggressive type of non-Hodgkins lymphoma.

25 Sep 2009

Recipients of the Lundbeck Neonatal Nursing Career Path Scholarship announced

The National Association of Neonatal Nurses (NANN) is proud to announce the recipients of the Lundbeck Neonatal Nursing Career Path Scholarship. The program, made possible through a grant from Lundbeck Inc. (formerly Ovation Pharmaceuticals), supports clinical neonatal nurses who seek to advance their education and become neonatal nurse practitioners.

25 Sep 2009

Drug trial for OSI-906 begins

TGen Clinical Research Services at Scottsdale Healthcare today announced the start of a clinical trial for a drug designed to combat adrenocortical carcinoma (ACC), a rare but deadly cancer that attacks the adrenal glands.

23 Sep 2009

NIH, NephCure Foundation and University of Michigan support study of nephrotic syndrome

University of Michigan Health System researchers will lead a $10.25 million effort to study rare kidney diseases that cause kidney failure and the need for dialysis.

23 Sep 2009

Neurodegenerative disorders are the second-leading cause of early-onset dementia, says report

Neurodegenerative disorders called frontotemporal dementias (FTD) are the second-leading cause of early-onset dementia in persons under age 65 and can emerge sometimes as early as age 20.

21 Sep 2009

New legislation protects healthcare coverage for rare disease clinical trial participants

Four members of the United States Senate introduced legislation today to allow patients with rare diseases to participate in clinical drug studies without losing their eligibility for public healthcare coverage, echoing a move by the House of Representatives last month.

17 Sep 2009

FDA provides Orphan-Drug designation for Keryx' KRX-0401 to treat multiple myeloma

Keryx Biopharmaceuticals, Inc. today announced that KRX-0401 (perifosine) has received Orphan-Drug designation from the U.S. Food and Drug Administration (FDA) for the treatment of multiple myeloma. In August, the Company announced that it had reached an agreement with the FDA regarding a Special Protocol Assessment (SPA) on the design of a Phase 3 trial in relapsed/refractory multiple myeloma and that the study is expected to start by year-end.

16 Sep 2009

Keryx Biopharmaceuticals receives orphan-drug designation for a multiple myeloma drug

AEterna Zentaris Inc., ("the Company") a global biopharmaceutical company focused on endocrine therapy and oncology, today announced that Keryx Biopharmaceuticals, Inc., its partner and licensee for perifosine in the North American market, has received orphan-drug designation for this compound from the U.S. Food and Drug Administration (FDA) for the treatment of multiple myeloma.

16 Sep 2009

FDA grants orphan drug status to Advanced Life Sciences' Restanza

Advanced Life Sciences Holdings, Inc. (OTC Bulletin Board: ADLS) today announced that the United States Food and Drug Administration (FDA) has granted Orphan Drug Designation to Restanza(TM) (cethromycin), its novel, once daily oral antibiotic, for the prophylactic treatment of plague and tularemia. Both tularemia and plague are classified by the Centers for Disease Control as Category A Bioterrorism Agents, which is the highest priority classification.

16 Sep 2009

NORD urges members of Congress to pass comprehensive health reform legislation

On behalf of the 30 million Americans who are affected by one of the nearly 7,000 rare disorders, the National Organization for Rare Disorders (NORD) urges you to pass comprehensive health reform legislation to assure that every American has access to affordable, meaningful health insurance coverage. Failure to pass such long-overdue legislation this year would be devastating for people with rare diseases, and for millions of others burdened by chronic illness.

15 Sep 2009

Rare Disease News and Research

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

CSL Behring LEAD program invites grant requests from organizations assisting people with rare diseases

Daily doses of imatinib mesylate double survival rates in Ph+ ALL affected children

Research to explore diagnosis and treatment of rare diseases

IDF announces new social networking online website for people with PIDD

The EURORDIS and NORD rare disease organizations form a partnership

Post Baccalaureate Premedical Program to begin in January 2010

Combined immunodeficiency results in severe health problems

Folotyn approved for the treatment of Peripheral T-Cell Lymphoma

Recipients of the Lundbeck Neonatal Nursing Career Path Scholarship announced

Drug trial for OSI-906 begins

NIH, NephCure Foundation and University of Michigan support study of nephrotic syndrome

Neurodegenerative disorders are the second-leading cause of early-onset dementia, says report

New legislation protects healthcare coverage for rare disease clinical trial participants

FDA provides Orphan-Drug designation for Keryx' KRX-0401 to treat multiple myeloma

Keryx Biopharmaceuticals receives orphan-drug designation for a multiple myeloma drug

FDA grants orphan drug status to Advanced Life Sciences' Restanza

NORD urges members of Congress to pass comprehensive health reform legislation

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