Rare Disease News and Research

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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

Repligen's RG2833 granted orphan drug designation for treatment of Friedreich's ataxia

Repligen Corporation announced today that the Office of Orphan Products Development of the Food and Drug Administration has granted orphan drug designation to RG2833, a selective histone deacetylase 3 inhibitor for the treatment of Friedreich's ataxia. Orphan drug designation qualifies Repligen to receive seven years of marketing exclusivity in the United States if the company is the first to obtain marketing approval for RG2833 for the treatment of Friedreich's ataxia.

24 May 2010

OnePath Gaucher Application available for patients with Type 1 Gaucher disease

Shire plc, the global specialty biopharmaceutical company, today announced the availability of the OnePath Gaucher Application. The first and only application developed specifically for patients in the U.S. with Type 1 Gaucher disease, it is free and ready for download at the iTunes Application store.

24 May 2010

Grifols reaches agreement with Pharmalink to acquire PPS drug development project

Today, Grifols SA announced that it has reached an agreement with the Swedish company Pharmalink AB to acquire various forms of intellectual property associated with the treatment of post-polio syndrome. The acquisition is expected to be finalized in the next few weeks and will include documentation, know-how, and Swedish regulatory approvals under the trade name Xepol.

20 May 2010

FDA grants orphan drug designation to pralatrexate for treatment of advanced TCC of urinary bladder

Allos Therapeutics, Inc. announced today that the U.S. Food and Drug Administration has granted orphan drug designation to pralatrexate for the treatment of advanced or metastatic transitional cell carcinoma of the urinary bladder, a form of bladder cancer. The Company is currently investigating pralatrexate in a Phase 2 clinical study in patients with advanced or metastatic relapsed TCC of the urinary bladder.

18 May 2010

FDA accepts for filing Sigma-Tau Pharmaceuticals' cysteamine hydrochloride ophthalmic solution NDA

Sigma-Tau Pharmaceuticals, Inc. announced today that the U.S. Food and Drug Administration (FDA) has accepted the filing of the company's new drug application (NDA) for its cysteamine hydrochloride ophthalmic solution, an investigational therapy for the treatment of corneal cystine crystals in patients with cystinosis. The FDA also granted the NDA Priority Review status, a designation given to drugs that may provide major advances or a treatment where no adequate therapy exists.

18 May 2010

Duchenne Muscular Dystrophy Awareness Week declared to honor work of PPMD

The Pennsylvania House of Representatives has passed a resolution declaring the week of May 24, 2010 as "Duchenne Muscular Dystrophy Awareness Week" to help honor the work of Parent Project Muscular Dystrophy, the largest non-profit organization in the United States focused on finding a cure for Duchenne muscular dystrophy.

15 May 2010

NIAMS, NINDS announce 5-year, $7.5M natural history study of Duchenne muscular dystrophy

The National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) and the National Institute of Neurological Disorders and Stroke (NINDS) are launching a five-year, $7.5 million natural history study of Duchenne muscular dystrophy (Duchenne), a degenerative genetically-linked neuromuscular disease. The study aims to validate non-invasive approaches to monitor the progression and treatment of Duchenne, and holds potential to facilitate the development of promising new therapies for people with the disease.

10 May 2010

Aeterna Zentaris granted FDA orphan-drug designation for AEZS-108 in ovarian cancer

Aeterna Zentaris Inc. a late-stage drug development company specialized in oncology and endocrinology, today announced that it has received from the U.S. Food and Drug Administration (FDA), orphan-drug designation for AEZS-108, its doxorubicin targeted conjugate compound, for the treatment of ovarian cancer. AEZS-108 is currently in a Phase 2 trial in advanced ovarian and advanced endometrial cancer in Europe.

6 May 2010

NxStage Medical first-quarter net revenue increases 20% to $40.4 million

NxStage Medical, Inc., a leading manufacturer of innovative dialysis products, today reported financial results for the first quarter of 2010 ended March 31, 2010, with total revenue above the top end of its guidance range.

5 May 2010

Triple-S Management first-quarter total consolidated operating revenues increase 9.8% to $519.1 million

Triple-S Management Corporation, the largest managed care company in Puerto Rico, today announced consolidated operating revenues of $519.1 million and operating income of $16.4 million for the three months ended March 31, 2010. Net income of $11.2 million, or $0.38 per diluted share, includes an after tax net gain of $0.4 million, or $0.01 per share, in net realized and unrealized gains on investments and derivatives.

5 May 2010

Cadence Pharmaceuticals resubmits OFIRMEV NDA

Cadence Pharmaceuticals, Inc. announced today that it has resubmitted its New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for its investigational product candidate, OFIRMEV™ (acetaminophen) injection, for the treatment of pain and fever in adults and children.

5 May 2010

LightLab Imaging granted FDA clearance for C7-XR Imaging System and C7 Dragonfly Imaging Catheter

LightLab Imaging, Inc., the pioneer and leader in the development of Optical Coherence Tomography (OCT) technology for vascular and other imaging applications, has received clearance from the U.S. Food and Drug Administration (FDA) for the C7-XR Imaging System and companion C7 Dragonfly Imaging Catheter.

5 May 2010

Micromet reports total revenues of $6.3 million for first-quarter 2010

Micromet, Inc., a biopharmaceutical company focused on the development and commercialization of next-generation antibodies for the treatment of cancer, today announced its financial results for the first quarter ended March 31, 2010.

5 May 2010

BELLUS Health to discontinue NC-503 diabetes development program

BELLUS Health announced today that it will discontinue its NC-503 diabetes development program following the results of the recently completed Phase IIa clinical proof-of-concept study evaluating NC-503 (eprodisate disodium) as a treatment for Type 2 diabetes. The study did not meet its primary efficacy endpoint.

5 May 2010

Rare Disease News and Research

NADHS organizes second annual Oral Cancer walk

BioSante's GVAX CML Vaccine granted FDA Orphan Drug designation

Millennium announces data on VELCADE in treatment of patients with amyloidosis

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

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

New model reveals molecular basis of multiple hereditary exostoses

Repligen's RG2833 granted orphan drug designation for treatment of Friedreich's ataxia

OnePath Gaucher Application available for patients with Type 1 Gaucher disease

Grifols reaches agreement with Pharmalink to acquire PPS drug development project

FDA grants orphan drug designation to pralatrexate for treatment of advanced TCC of urinary bladder

FDA accepts for filing Sigma-Tau Pharmaceuticals' cysteamine hydrochloride ophthalmic solution NDA

Duchenne Muscular Dystrophy Awareness Week declared to honor work of PPMD

NIAMS, NINDS announce 5-year, $7.5M natural history study of Duchenne muscular dystrophy

Aeterna Zentaris granted FDA orphan-drug designation for AEZS-108 in ovarian cancer

NxStage Medical first-quarter net revenue increases 20% to $40.4 million

Triple-S Management first-quarter total consolidated operating revenues increase 9.8% to $519.1 million

Cadence Pharmaceuticals resubmits OFIRMEV NDA

LightLab Imaging granted FDA clearance for C7-XR Imaging System and C7 Dragonfly Imaging Catheter

Micromet reports total revenues of $6.3 million for first-quarter 2010

BELLUS Health to discontinue NC-503 diabetes development program

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