Rare Disease News and Research

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Enobia completes patient enrollment in ENB-0040 Phase II study for HPP

Enobia Pharma Inc., today announced that it has successfully completed enrollment in its Phase II study of ENB-0040, a bone targeted enzyme replacement therapy being investigated in adolescents and adults with hypophosphatasia, a serious, rare metabolic bone disorder.

25 Feb 2011

Study: Rare genetic mutations may provide clues to improve treatment for schizophrenia

Scientists are eyeing a rare genetic glitch for clues to improved treatments for some people with schizophrenia - even though they found the mutation in only one third of 1 percent of patients.

25 Feb 2011

PHA partners with NORD, EURORDIS to raise global awareness about PH

Pulmonary hypertension is a rare, under-diagnosed disease that can strike people at any time, affecting newborns to the elderly or anyone in between. To raise global awareness of this invisible disease, the Pulmonary Hypertension Association is partnering with the National Organization for Rare Disorders and Rare Diseases Europe on Feb. 28, 2011, to observe Rare Disease Day.

25 Feb 2011

PhRMA: 460 medicines in development for rare diseases

America's biopharmaceutical research companies are intensifying research into rare diseases, which often are among the most devastating to patients and complex for researchers. A record 460 medicines for rare diseases are in late stages of the pipeline, either in clinical trials or awaiting Food and Drug Administration (FDA) review, according to a report released today by the Pharmaceutical Research and Manufacturers of America (PhRMA).

24 Feb 2011

FDA grants Fast Track designation to Sunesis' vosaroxin for treatment of AML

Sunesis Pharmaceuticals, Inc. today announced that vosaroxin, the Company's lead drug candidate, has been granted Fast Track designation by the U.S. Food and Drug Administration for the potential treatment of relapsed or refractory acute myeloid leukemia in combination with cytarabine.

23 Feb 2011

Macrogen announces formation of Axeq Technologies in the US, Europe and Asia

Macrogen, Inc announces the formation of Axeq Technologies, a US-based division with operations and offices in the US, Europe and Asia. "Axeq" is a premium brand representing a whole new experience and industry-leading standards in DNA sequencing service.

22 Feb 2011

U.S. FDA approves Corifact to prevent bleeding in people with Factor XIII deficiency

The U.S. Food and Drug Administration today approved Corifact, the first product intended to prevent bleeding in people with the rare genetic defect congenital Factor XIII deficiency.

18 Feb 2011

FDA grants miRagen's MGN-4893 orphan drug designation for treatment of polycythemia vera

miRagen Therapeutics, Inc., a privately-held biopharmaceutical company focused on improving patients' lives by developing innovative microRNA (miRNA)-based therapeutics for cardiovascular and muscle disease, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to the company's compound MGN-4893 for the treatment of polycythemia vera, a myeloproliferative disease characterized by an overabundance of blood cells and platelets in the body.

18 Feb 2011

Sanofi-aventis enters definitive agreement to acquire Genzyme

Sanofi-aventis and Genzyme Corporation announced today that they have entered into a definitive agreement under which sanofi-aventis is to acquire Genzyme for $74.00 per share in cash, or approximately $20.1 billion.

16 Feb 2011

NHGRI's new strategic plan to identify genetic bases of most single-gene disorders

A new strategic plan from an arm of the National Institutes of Health envisions scientists being able to identify genetic bases of most single-gene disorders and gaining new insights into multi-gene disorders in the next decade. This should lead to more accurate diagnoses, new drug targets and the development of practical treatments for many who today lack therapeutic options, according to the plan from the National Human Genome Research Institute (NHGRI).

14 Feb 2011

Alexion fourth quarter net product sales of Soliris increases to $156.0 million

Alexion Pharmaceuticals, Inc. today announced financial results for the quarter and year ended December 31, 2010. For the three months ended December 31, 2010, Alexion Pharmaceuticals, Inc. reported net product sales of Soliris® (eculizumab) of $156.0 million, reflecting strong additions of new patients, compared to $110.6 million for the same period in 2009.

10 Feb 2011

FDA grants Neuralstem orphan drug designation for treatment of ALS with spinal cord stem cells

Neuralstem, Inc. announced that the U.S. Food and Drug Administration's Office of Orphan Products Development has granted it orphan drug designation for the treatment of Amyotrophic Lateral Sclerosis (ALS) with its human spinal cord derived neural stem cells (NSI-566RSC), currently in a Phase I safety study to evaluate the safety of the product and the surgical route of administration in a wide range of ALS patients.

10 Feb 2011

Everolimus phase III study shows improved progression-free survival in patients with pNET

In an international Phase III randomized study, everolimus, an inhibitor of the mammalian target of rapamycin, has shown to dramatically improve progression-free survival for patients with advanced pancreatic neuroendocrine tumors, according to researchers from The University of Texas MD Anderson Cancer Center.

9 Feb 2011

Lundbeck announces second annual campaign to raise awareness on rare diseases

Today, Lundbeck announced its second annual Raise Your Hand to Fight Rare Diseases campaign in support of Rare Disease Day, a worldwide observance each year on February 28 to raise awareness of rare diseases.

8 Feb 2011

New understanding of how host and foreign structures are distinguished by front-line defence mechanism

This week, the highly-respected US Academy of Sciences journal (PNAS) published an article describing how the first line of defence of the human immune system distinguishes between microbes and the body's own structures.

5 Feb 2011

How body's first line of defence distinguishes between microbes and own structures

4 Feb 2011

Rare new disease identified – calcium accumulates in blood vessels

A new rare disease has been identified. Louise Benge’s started having difficulty in walking when she was 25. She developed hardening and pain over her calves and hands. She worsened over the next 20 years. Her family physician in Mount Vernon, Ky., was at a loss, as were a vascular specialist, a hand specialist and a kidney specialist. Her two sisters and two brothers had the problem too, but no doctor could understand why.

3 Feb 2011

$FDA grants Onyx fast track designation for carfilzomib in treatment of relapsed and refractory multiple myeloma$

FDA grants Onyx fast track designation for carfilzomib in treatment of relapsed and refractory multiple myeloma

Onyx Pharmaceuticals, Inc. announced today that the U.S. Food and Drug Administration (FDA) has granted fast track designation for carfilzomib, a next generation proteasome inhibitor for the potential treatment of patients with relapsed and refractory multiple myeloma.

1 Feb 2011

FDA schedules fourth orphan drug designation workshop

The U.S. Food and Drug Administration has scheduled its fourth orphan drug designation workshop for academics, biotechnology companies, and those unfamiliar with the process for Feb. 28 – March 1, 2011, in Claremont, Calif. in collaboration with Keck Graduate Institute.

28 Jan 2011

FDA grants BioSante Orphan Drug designation for Melanoma Cancer Vaccine

BioSante Pharmaceuticals, Inc. today announced the receipt of Orphan Drug designation for its Melanoma Cancer Vaccine in the treatment of stage IIb to IV melanoma, from the FDA's Office of Orphan Products Development. Melanoma is the most deadly form of skin cancer.

25 Jan 2011

Rare Disease News and Research

Enobia completes patient enrollment in ENB-0040 Phase II study for HPP

Study: Rare genetic mutations may provide clues to improve treatment for schizophrenia

PHA partners with NORD, EURORDIS to raise global awareness about PH

PhRMA: 460 medicines in development for rare diseases

FDA grants Fast Track designation to Sunesis' vosaroxin for treatment of AML

Macrogen announces formation of Axeq Technologies in the US, Europe and Asia

U.S. FDA approves Corifact to prevent bleeding in people with Factor XIII deficiency

FDA grants miRagen's MGN-4893 orphan drug designation for treatment of polycythemia vera

Sanofi-aventis enters definitive agreement to acquire Genzyme

NHGRI's new strategic plan to identify genetic bases of most single-gene disorders

Alexion fourth quarter net product sales of Soliris increases to $156.0 million

FDA grants Neuralstem orphan drug designation for treatment of ALS with spinal cord stem cells

Everolimus phase III study shows improved progression-free survival in patients with pNET

Lundbeck announces second annual campaign to raise awareness on rare diseases

New understanding of how host and foreign structures are distinguished by front-line defence mechanism

How body's first line of defence distinguishes between microbes and own structures

Rare new disease identified – calcium accumulates in blood vessels

FDA grants Onyx fast track designation for carfilzomib in treatment of relapsed and refractory multiple myeloma

FDA schedules fourth orphan drug designation workshop

FDA grants BioSante Orphan Drug designation for Melanoma Cancer Vaccine

Industry Focus eBook - Cell & Gene Therapy (1st Edition) eBook

Designing fluidic architecture for scalable diagnostic platforms

Evaluating new approaches for rare disease genomics

Expanding access to NMR with the Fourier 80 Duo

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