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Researchers identify a set of molecular signatures for biliary atresia

Researchers have identified a set of "molecular signatures" for biliary atresia - the most common diagnosis leading to liver transplant in children - that can help identify the progression of disease at diagnosis and predict clinical outcomes.

1 Jul 2010

NORD urges FDA to reduce regulatory uncertainty in developing medicines for rare disorders

Noting that only about 200 of the nearly 7,000 diseases classified as rare currently have treatments, the National Organization for Rare Disorders (NORD) today called for a Food and Drug Administration (FDA) statement of policy on regulation of therapies for rare disorders.

30 Jun 2010

Recent, upcoming FDA efforts address rare, 'neglected' diseases

The FDA's new rare disease review group will hold its first public hearing on Tuesday and Wednesday to discuss how to expand efforts to develop treatments for rare diseases that affect fewer than 200,000 people, Reuters

30 Jun 2010

FDA grants t2cure orphan drug designation for t2c001 in Buerger's Disease

t2cure GmbH, a biopharmaceutical company developing and marketing stem cell-based regenerative therapies, announced today that the FDA has granted orphan drug designation to t2c001 for the treatment of Buerger's Disease. The Company also announced that in a concurrent application in Europe the EMA's Committee for Orphan Medicinal Products recently recommended t2c001 for orphan designation to the European Commission.

24 Jun 2010

FDA grants Tolera's TOL101 orphan drug designation for treatment of T1DM

Tolera Therapeutics, Inc., was granted orphan drug status by the United States Food and Drug Administration (FDA) for TOL101 (anti-TCR murine monoclonal antibody, type IgM), for treatment of recent onset immune-mediated Type 1 diabetes mellitus (T1DM). Designation is granted for treatment of patients sixteen years of age and younger with immune-mediated T1DM and preserved pancreatic beta-cell function, commonly referred to as juvenile diabetes.

23 Jun 2010

Teens to call upon Congress to fund drug research for rare diseases

Teens from across the United States whose siblings have cystic fibrosis will press their elected officials in Washington, D.C., June 24, to fund drug research and increase access to clinical trials for those with rare diseases.

22 Jun 2010

Dyax signs partnership agreement with Sigma-Tau to develop and commercialize subcutaneous DX-88

Dyax Corp. and Defiante Farmaceutica S.A., a subsidiary of the pharmaceutical company Sigma-Tau SpA, announced today a strategic partnership to develop and commercialize subcutaneous DX-88 (ecallantide) for the treatment of hereditary angioedema and other therapeutic indications throughout Europe, North Africa, Middle East and Russia.

21 Jun 2010

End Duchenne Day to be celebrated on June 26, 2010: PPMD

Patricia A. Furlong, Founding President and CEO of Parent Project Muscular Dystrophy, the largest non-profit organization in the United States focused on finding a cure for Duchenne muscular dystrophy, announced that the Mayor of Denver, Colorado, John W. Hickenlooper, has proclaimed June 26, 2010 "End Duchenne Day" in recognition of PPMD's 16th Annual Connect Conference. Denver is playing host to the country's largest Duchenne-specific, international conference, June 24 – 27, 2010.

19 Jun 2010

EC grants Allos Therapeutics' pralatrexate orphan medicinal product designation for CTCL treatment

Allos Therapeutics, Inc. announced today that the European Commission (EC) has granted orphan medicinal product designation for pralatrexate for the treatment of cutaneous T-cell lymphoma (CTCL). The Company is currently investigating pralatrexate in a Phase 1 clinical study in patients with relapsed or refractory CTCL.

18 Jun 2010

FDA seeks public input on regulation of laboratory-developed tests

The U.S. Food and Drug Administration today announced plans to hold a public meeting on July 19-20, 2010, to discuss how the agency will oversee laboratory-developed tests (LDTs).

17 Jun 2010

European Commission grants Orphan Medicinal Product Designation for QPI-1002

Quark Pharmaceuticals, Inc., a world leader in the discovery and development of RNAi-based therapeutics, today announced that the European Commission has granted Orphan Medicinal Product Designation for QPI-1002 (also referred to as "I5NP") for the prophylaxis of delayed graft function in kidney transplant patients.

16 Jun 2010

IMF concludes first-of-its-kind inaugural Myeloma Summit

The International Myeloma Foundation, the oldest and largest foundation dedicated to improving the life and care of myeloma patients, has concluded the first-of-its-kind inaugural Myeloma Summit meeting - nearly 70 of the world's leading experts in myeloma and related blood cancers charting the future of myeloma treatment and care.

14 Jun 2010

BIOTECanada congratulates CPHA on 100 years of leadership in public health

As a community partner of the Canadian Public Health Association, BIOTECanada, on behalf of Canada's biotechnology industry, congratulates the CPHA on 100 years of leadership in public health.

11 Jun 2010

FDA grants orphan drug designation for ICT-107

ImmunoCellular Therapeutics announced today that the U.S. Food and Drug Administration has granted orphan drug designation for ICT-107, the company's dendritic cell-based cancer vaccine candidate which targets glioblastoma multiforme. As a result of the orphan drug status, ImmunoCellular will be eligible to receive a number of benefits, including access to grant funding for clinical trials, tax credits, accelerated FDA approval and allowance for marketing exclusivity after drug approval for a period of up to seven years.

11 Jun 2010

NADHS organizes second annual Oral Cancer walk

For the second year, the Nashville Area Dental Hygienists' Society (NADHS) has organized a successful walk to promote oral cancer awareness for a disease that affects so many, yet so few know about. Hundreds of Nashville locals gathered for the "Boot Scootin' for Oral Cancer Screening II" event that recently took place at Nashville's Centennial Park to raise disease awareness, and funds for the Non-Profit Oral Cancer Foundation (OCF).

11 Jun 2010

BioSante's GVAX CML Vaccine granted FDA Orphan Drug designation

BioSante Pharmaceuticals, Inc. today announced the receipt of Orphan Drug designation for GVAX CML Vaccine in the treatment of chronic myeloid leukemia (CML) from the FDA's Office of Orphan Products Development. Orphan drug designation, as granted by the U.S. Orphan Drug Act, is for a product to treat a rare disease or condition that affects fewer than 200,000 people in the U.S. Orphan drug designation qualifies the sponsor of the product for tax credits and seven years of marketing exclusivity, among other benefits.

7 Jun 2010

Millennium announces data on VELCADE in treatment of patients with amyloidosis

Millennium: The Takeda Oncology Company today announced data examining the use of VELCADE in the treatment of patients with relapsed or refractory light chain (AL) amyloidosis. Amyloidosis is a rare condition in which excessive proteins build up in organs or tissues, such as the heart, kidney or liver. These data were presented at the Annual Meeting of the American Society of Clinical Oncology (ASCO) held in Chicago, Illinois, June 4-8, 2010.

5 Jun 2010

ASCO 2010 marks first anniversary of "Cancer Patient Statement of Principles"

The International Myeloma Foundation (IMF), the oldest and largest foundation dedicated to improving the life and care of myeloma patients, notes the 2010 Annual Meeting of the American Society for Clinical Oncology (ASCO) marks the first anniversary of the "Cancer Patient Statement of Principles."

4 Jun 2010

McGill University granted 'Orphan Drug Designation' for pulmonary infection drug fenretinide

In addition to their suffering, rare disease patients often have to face the harsh reality that few pharmaceutical companies will ever be able to offer new treatments for their condition because the costs of new treatments will never be recovered from such a small market. But there are ways they can be helped. The U.S. Food and Drug Administration's "Orphan Drug Designation" offers a wide range of benefits that help organizations developing treatments for diseases and conditions affecting fewer than 200,000 patients in the United States.

2 Jun 2010

New model reveals molecular basis of multiple hereditary exostoses

Children born with multiple hereditary exostoses (MHE) suffer from abnormal growths on their bones. These bony protrusions stunt their growth and can cause pain and disfigurement.

2 Jun 2010