Actelion CEO says new use may double Tracleer sales

Wednesday, August 27, 2008


  • Tracleer approval for pulmonary fibrosis could double sales
  • Company aims to remain independent
  • Not considering big acquisition
Aug 27  »   Actelion CEO says new use may double...

Actelion CEO says new use may double Tracleer sales

Wednesday, August 27, 2008


  • Tracleer approval for pulmonary fibrosis could double sales
  • Company aims to remain independent
  • Not considering big acquisition
Aug 27  »   Dr. Eleni Galanis Two cases of...

Dr. Eleni Galanis Two cases of Listeriosis discovered in Northwest as death toll rises

Wednesday, August 27, 2008

THE RECENT food borne illness that’s forced Maple Leaf Foods Canada to recall more than 20 of its products has reached the Northwest.
Aug 27  »   33 PV cases confirmed

33 PV cases confirmed

Wednesday, August 27, 2008

HAZLETON — The federal Agency for Toxic Substances and Disease Registry on Monday confirmed something that residents of the intersections of Schuylkill, Carbon and Luzerne counties have thought for years — an unusually high number of people there are suffering from a rare blood cancer.
Aug 27  »   Glivec receives FDA priority review as...

Glivec receives FDA priority review as first therapy to reduce recurrence of gastrointestinal stromal tumors after surgery

Wednesday, August 27, 2008

Clinical data showing unprecedented 89% reduction in risk of GIST relapse with use of Glivec after surgery are basis for FDA, EMEA, Swissmedic filings.
Aug 27  »   Rare disease strikes kids under 5

Rare disease strikes kids under 5

Wednesday, August 27, 2008

Retinoblastoma is a rare type of eye cancer that mostly affects children under the age of five.
Aug 27  »   Pharmalink reports positive phase III...

Pharmalink reports positive phase III results for Xepol®, a treatment for Post-Polio Syndrom

Wednesday, August 27, 2008

STOCKHOLM, SWEDEN – Pharmalink AB today announces positive results from a follow-on Phase III study of Xepol®, its candidate for the treatment of post-polio syndrome (PPS). The data have shown the candidate to be effective and well tolerated with no serious adverse events attributed to the product being reported in the treated patients.
Aug 27  »   Gentium announces Italian legal proceeding

Gentium announces Italian legal proceeding

Wednesday, August 27, 2008

VILLA GUARDIA, Italy, - Gentium S.p.A. (NASDAQ: GENT) (the "Company") today announced that a legal proceeding has been commenced against the Company's Board of Directors and Board of Statutory Auditors in the Court of Como in Italy seeking to prevent a proposed financing transaction from proceeding and alleging misconduct in connection with that transaction.
Aug 26  »   New oral vaccine may protect against...

New oral vaccine may protect against Bubonic Plague

Tuesday, August 26, 2008

Researchers from the Institut Pasteur in Paris, France used a less virulent ancestor to the highly infectious bubonic plague to develop a potentially safe, efficient and inexpensive live oral vaccine. They report their findings in the August 2008 issue of the journal Infection and Immunity.
Aug 26  »   Genzyme Corporation and Medicines for...

Genzyme Corporation and Medicines for Malaria Venture Announce New Collaboration with Advinus Therapeutics to Accelerate Discovery of Novel Therapies for Malaria

Tuesday, August 26, 2008


Brings complementary capabilities in India to address patient populations most at risk
Aug 26  »   Autism's cause remains mystery

Autism's cause remains mystery

Tuesday, August 26, 2008

Pediatricians do not know what causes autism or autism spectrum disorders, but we do understand the frustration of families who want to know the answers.
Aug 26  »   BioMarin announces roll-out of national...

BioMarin announces roll-out of national PKU registry

Tuesday, August 26, 2008

NOVATO, Calif. - BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) announced today the roll-out of the national PKU Demographics, Outcomes and Safety Registry (PKUDOS). The registry is open to all PKU clinics within the United States, and all patients screened for Kuvan responsiveness are eligible for enrollment in the registry.
Aug 26  »   For Rare Blood Disorder, Pumping Iron Is...

For Rare Blood Disorder, Pumping Iron Is the Cure

Tuesday, August 26, 2008

Bloodletting is often dismissed as a primitive form of medicine, in which early doctors attempted to rid the body of bad humors by draining the life-sustaining fluid.
Aug 26  »   Leading neurologist opens Missouri...

Leading neurologist opens Missouri Baptist Medical Center's first MS Center

Tuesday, August 26, 2008

ST. LOUIS, - Barry Singer, M.D., leading neurologist and adjunct assistant professor of clinical neurology at Washington University School of Medicine, will open The MS Center for Innovations in Care on the campus of Missouri Baptist Medical Center on August 18th.
Aug 26  »   FDA requires additional information on...

FDA requires additional information on DORIBAX for treatment of Hospital-Acquired Pneumonia

Tuesday, August 26, 2008

RARITAN, N.J. - Johnson & Johnson Pharmaceutical Research & Development, L.L.C. (J&JPRD) today announced that the U.S. Food and Drug Administration (FDA) requires additional information before it will approve the company's New Drug Application (NDA) for DORIBAX(TM) (doripenem for injection) for the treatment of hospital-acquired pneumonia, also known as nosocomial pneumonia...
Aug 26  »   Synta announces publication of Elesclomol...

Synta announces publication of Elesclomol mechanism of action results in AACR Journal Molecular Cancer Therapeutics

Tuesday, August 26, 2008


Oxidative stress induction triggers cancer cell death
Aug 25  »   The Multiple Myeloma Research Consortium...

The Multiple Myeloma Research Consortium (MMRC) partners with Celgene Corporation on phase I/II trial of Pomalidomide (CC-4047) in relapsed and refractory Multiple Myeloma patients

Monday, August 25, 2008

The Multiple Myeloma Research Consortium (MMRC) today announced the initiation of a clinical trial with the oral immunomodulatory agent pomalidomide (CC-4047) in patients who have received at least two prior therapies including treatments with REVLIMID(R) (lenalidomide) and VELCADE(R) (bortezomib) for Injection.
 
 
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Top CheckOrphan Articles

 »   The girls with too much skin

The girls with too much skin

Research into a rare genetic disorder which makes sufferers grow more skin in one day than a healthy person does in two weeks has identified a mutation which may lead to development of new treatments.
 »   Autistic children 'should be given Prozac...

Autistic children 'should be given Prozac to control their symptoms'

More autistic children in the UK should be given drugs like Prozac to control their symptoms, an expert said today.
 »   'I will always feed you'

'I will always feed you'


The story of a father and son and their battle against a debilitating disorder
 »   A drug with efficacy superior to that of...

A drug with efficacy superior to that of remicade at inducing closure and healing of fistulas would earn a 30 percent patient share in the Crohn's disease drug market


WALTHAM, Mass. - Decision Resources, one of the world's leading research and advisory firms focusing on pharmaceutical and healthcare issues, finds that a therapy that induces closure and healing of fistulas in a greater percentage of patients than Centocor/Schering-Plough/Mitsubishi Tanabe's Remicade (infliximab) does, would earn a 30 percent patient share in the treatment of Crohn's...
 »   Genzyme rises on prediction of profit growth

Genzyme rises on prediction of profit growth

NEW YORK - Shares of Genzyme Corp. moved higher Wednesday after an analyst upgraded shares of the biotechnology company, saying sales of three key products will help the company reach its growth targets over the next five years.
 »   Immunomedics, Inc. (IMMU)'s Milatuzumab...

Immunomedics, Inc. (IMMU)'s Milatuzumab receives FDA orphan drug designation

Immunomedics, Inc., (NasdaqGM:IMMU), a biopharmaceutical company focused on developing monoclonal antibodies to treat cancer and other serious diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to milatuzumab for the treatment of multiple myeloma.
 »   UNICEF & Merck partner to protect 100,000...

UNICEF & Merck partner to protect 100,000 children and young people against mumps epidemic


NEW YORK - Officials with the U.S. Fund for UNICEF and global pharmaceutical company, Merck & Co., Inc. announced the donation and delivery of 100,000 doses of M-M-R(R) II (measles, mumps and rubella virus vaccine, live, attenuated) to the government of the former Soviet republic of Moldova, in response to an ongoing serious mumps outbreak in that country.
 »   CEL-SCI presents favorable data for LEAPS...

CEL-SCI presents favorable data for LEAPS vaccine technology at prestious immunology conference


VIENNA, VA - CEL-SCI Corporation (AMEX: CVM) and researchers at Northeastern Ohio Universities Colleges of Medicine and Pharmacy jointly announced favorable new developments in the LEAPS™ heteroconjugate vaccine technology developed by CEL-SCI at the FOCIS Immunology meeting in Boston, MA on June 8, 2008. Patricia Taylor, M.S., working with Dr. Ken S. Rosenthal, Professor of...
 »   AVI BioPharma to present at the 6th...

AVI BioPharma to present at the 6th Annual Biodefense Vaccines and Therapeutics Meeting


PORTLAND, OR - AVI BioPharma, Inc. (NASDAQ: AVII) today announced that Patrick Iversen, Ph.D., AVI’s Senior Vice President of Strategic Alliances, will make the opening presentation of the Pre–Conference Symposium to the 6th Annual Biodefense Vaccines & Therapeutics Meeting to be held June 9–11, 2008 in Washington, D.C. The Symposium is titled “Developments in Biodefense Vaccines...
 »   Parents' fight to protect their cotton...

Parents' fight to protect their cotton wool boy

Some parents would like to keep their children in cotton wool, seeking to shelter them from life’s hard knocks. Others simply do not have a choice. At 21 months, Caloundra’s Lachlan Woodward is a sweet, strong-willed and active little boy, eager to explore his surroundings and test the boundaries.

Rare diseases to be main theme at 11th European Health Forum

Wednesday, August 27, 2008

It is slowly becoming a permanent establishment for the Gasteiner valley. The 2008 European Health Forum, the most important health politics conference in Europe, will take place this year once again in Bad Hofgastein, for the eleventh time. The forum will begin on Wednesday, October 1, and continue through Saturday, October 4.
Aug 27  »   Rare diseases to be main theme at 11th...

Rare diseases to be main theme at 11th European Health Forum

Wednesday, August 27, 2008

It is slowly becoming a permanent establishment for the Gasteiner valley. The 2008 European Health Forum, the most important health politics conference in Europe, will take place this year once again in Bad Hofgastein, for the eleventh time. The forum will begin on Wednesday, October 1, and continue through Saturday, October 4.
Aug 27  »   Research examines variations of rare lung...

Research examines variations of rare lung disease

Wednesday, August 27, 2008

CINCINNATI - Lymphangioleiomyomatosis, or LAM, is a rare but serious lung disease that may cause severe respiratory symptoms in patients. The often-fatal disease has no cure.
Aug 27  »   Pioneering Neurosciences Institute sets...

Pioneering Neurosciences Institute sets date for dedication of new facility and first-ever international forum on memory and memory disorders

Wednesday, August 27, 2008

On October 16 world-renowned scientists will convene to share cutting edge research on Alzheimer's Disease, memory and aging.
Aug 27  »   Two experiments suggest new direction...

Two experiments suggest new direction for diabetes

Wednesday, August 27, 2008


WASHINGTON (Reuters) - Two experimental treatments suggest new directions for treating diabetes, both using compounds already made by the body, researchers in the United States reported on Monday.
Aug 27  »   History of nonmelanoma skin cancer is...

History of nonmelanoma skin cancer is associated with increased risk for subsequent malignancies

Wednesday, August 27, 2008

Aug 27  »   Angiotensin inhibitors and receptor...

Angiotensin inhibitors and receptor blockers linked to lower risk of nonmelanoma skin cancer

Wednesday, August 27, 2008

The use of angiotensin-converting enzyme (ACE) inhibitors or angiotensin receptor blockers (ARBs) was associated with a reduced risk of basal cell or squamous cell skin cancers in U.S. veterans, researchers report in the August 26 online issue of the Journal of the National Cancer Institute.
Aug 27  »   Consumption of nuts, corn or popcorn not...

Consumption of nuts, corn or popcorn not associated with increased risk of diverticulosis in men

Wednesday, August 27, 2008

Contrary to a common recommendation to avoid eating popcorn, nuts and corn to prevent diverticular complications, a large prospective study of men indicates that the consumption of these foods does not increase the risk of diverticulosis or diverticular complications, according to a study in the August 27 issue of JAMA.
Aug 27  »   Unusual ultrasonic vocalization patterns...

Unusual ultrasonic vocalization patterns in mice may be useful for modeling autism

Wednesday, August 27, 2008

Scientists have found novel patterns of ultrasonic vocalizations in a genetic mouse model of autism, adding a unique element to the available mouse behaviors that capture components of the human disease, and representing a new step towards identifying causes and better treatments.
Aug 26  »   Eculizumab’s triumph over PNH

Eculizumab’s triumph over PNH

Tuesday, August 26, 2008

The FDA approval of the orphan drug eculizumab in March 2007 marked a distinct change in how physicians were able to treat patients with paroxysmal nocturn-al hemoglobinuria.
Aug 26  »   Gene that causes Childhood Cancer...

Gene that causes Childhood Cancer Neuroblastoma is found

Tuesday, August 26, 2008


May Lead to New Prevention and Treatment Strategies
Aug 26  »   Computer-Based method IDs Alzheimer's...

Computer-Based method IDs Alzheimer's protein structures

Tuesday, August 26, 2008

FRIDAY, Aug. - A new method of identifying protein structures related to Alzheimer's disease has been developed by researchers at the Massachusetts Institute of Technology.
Aug 26  »   Cancer cluster confirmed in northeast...

Cancer cluster confirmed in northeast Pennsylvania

Tuesday, August 26, 2008

HAZLETON, Pa. (AP) - A federal agency admits there's a 20-mile stretch in Pennsylvania where residents have an elevated risk of contracting a rare blood cancer.
Aug 26  »   Explaining a genetic disorder's unique shift

Explaining a genetic disorder's unique shift

Tuesday, August 26, 2008

Findings reported in this week's PLoS Biology give insight into the unique characteristics of the birth defect known as Prader-Willi Syndrome (PWS), and at the same time, may help explain the way that a certain type of gene is expressed in all humans.
Aug 26  »   Extensively drug-resistant tuberculosis...

Extensively drug-resistant tuberculosis can be managed with aggressive treatment strategy

Tuesday, August 26, 2008

The global threat of extensively drug-resistant tuberculosis (XDR TB) could be managed with aggressive treatment programmes, reducing the mortality associated with the condition and preventing further transmission. These are the conclusions of an Article published early Online and in an upcoming edition of The Lancet, authored by Dr Salmaan Keshavjee, Department of Global Health and Social...
Aug 26  »   New findings explain genetic disorder's...

New findings explain genetic disorder's unique shift

Tuesday, August 26, 2008

KNOXVILLE - Findings reported in this month's issue of PLoS Biology give insight into the unique characteristics of the birth defect known as Prader-Willi Syndrome (PWS), and at the same time, may help explain the way that a certain type of gene is expressed in all humans.
Aug 25  »   Alzheimer's brain dye separates the...

Alzheimer's brain dye separates the forgetful from the doomed

Monday, August 25, 2008

On some mornings Rose Chuderewicz, 80, can't remember how to put on her bra. She writes notes to remind herself to do daily tasks, then forgets to read them.
 
 
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Bullseye – shooting with Erythema nodosum

Wednesday, August 27, 2008


Eight years ago, Sonja Nowakoski from Moers, Germany, became almost completely blind. She thought she would never again participate in sports. Today, she is a successful sport shooter on the way to becoming the German champion.
Aug 27  »   Bullseye – shooting with Erythema nodosum

Bullseye – shooting with Erythema nodosum

Wednesday, August 27, 2008


Eight years ago, Sonja Nowakoski from Moers, Germany, became almost completely blind. She thought she would never again participate in sports. Today, she is a successful sport shooter on the way to becoming the German champion.
Aug 27  »   Condition only skin-deep

Condition only skin-deep

Wednesday, August 27, 2008

PIERSON -- Matt Land has always stood out.

As a youngster, he stood out because he physically looked different from everyone else.
Aug 27  »   Big-hearted Eels help out

Big-hearted Eels help out

Wednesday, August 27, 2008

Milliarn Lopez, who's nearly two, has found herself in the spotlight during many of the Parramatta Eels' home games.
Aug 27  »   ELA to receive UEFA cheque

ELA to receive UEFA cheque

Wednesday, August 27, 2008


UEFA has awarded its annual CHF 1m (€619,000) charity cheque to the European Leukodystrophy Association (ELA).
Aug 27  »   Rare disease found in Nevada woman

Rare disease found in Nevada woman

Wednesday, August 27, 2008

Tularemia, a rare disease often found in rabbits and rodents, has infected a west-central Nevada woman, according to health reports.
Aug 27  »   McKiernan doing well

McKiernan doing well

Wednesday, August 27, 2008

Ennis soldier back on track after battle with nerve disease.
Aug 26  »   Lowe Syndrome – towards a molecular...

Lowe Syndrome – towards a molecular understanding

Tuesday, August 26, 2008

Diseases often arise due to genetic changes. Lowe Syndrome is an example of this. It is caused by mutations in a gene called OCRL. Now researchers at Harvard University have discovered how these changes lead to the debilitating and life-threatening disease symptoms.
Aug 26  »   Living with a creeping paralysis

Living with a creeping paralysis

Tuesday, August 26, 2008


Six months ago, Sabine Menassa-Matta was struck down with a rare but dramatic neurological disease.
Aug 26  »   Redefining health: Writer reveals...

Rede