Hereditary in Medical Technology

Scientific Data Relating to Cinryze(TM) Presented at 6th Annual C1 Inhibitor Deficiency Workshop

... in October 2008 for routine prophylaxis against hereditary angioedema (HAE) attacks in adults and ... page 27: Prophylactic Therapy Considerations in hereditary Angioedema In an abstract ... Should Prophylactic Therapy be Considered for hereditary Angioedema?', Dr. Tim Craig, et al. described ...

FDA Grants Priority Review of a Supplemental Biologics License Application for Cinryze(TM) C1 Inhibitor (Human) as Treatment for Acute Attacks of Hereditary Angioedema (HAE)

... (human) as a treatment for acute attacks of hereditary Angioedema (HAE). The supplemental Biologics ... to the patients who suffer acute attacks of hereditary angioedema," commented Vincent Milano, ... an injection rate of 1 mL per minute. About hereditary Angioedema HAE is a rare, severely ...

ViroPharma Announces Presentation of Cinryze(TM) Data in Acute Treatment of Hereditary Angioedema

... successfully treated all types of acute hereditary angioedema (HAE) attacks seen in the study ... entitled "Shortcomings in the diagnosis of hereditary angioedema: Are we adequately screening family ... a challenge, and despite the fact that HAE is a hereditary disorder, testing family members is not common ...

ViroPharma Incorporated Completes Acquisition of Lev Pharmaceuticals

... Exclusivity for Prophylaxis against hereditary Angioedema - EXTON, Pa., Oct. 21 ... attacks in adolescent and adult patients with hereditary angioedema (HAE). hereditary angioedema, or C1 esterase inhibitor (C1-INH) ...

Changing Paradigms in Hereditary Angioedema: A Focus on Prompt Diagnosis and New Therapies

... for Healthcare Professionals VOORHEES, N.J., March 17 /PRNewswire/ -- hereditary angioedema (HAE) is estimated to afflict 10,000 people in the United ... States. This continuing education activity, titled Changing Paradigms in hereditary Angioedema: A Focus on Prompt Diagnosis and New Therapies, is supported ...

Shire to add New Orphan Drug to its HGT Portfolio - EU Launch Imminent

... company, Jerini AG for an equity purchase price of EUR328 million adding hereditary angioedema (HAE) product, FIRAZYR(R), to its Human Genetic Therapies ... stability. FIRAZYR will be packaged in a pre-filled syringe. About hereditary Angioedema (HAE) HAE is a debilitating and potentially life-threatening ...

AlphaMed Pharmaceuticals Announces Breakthrough in the Development of Recombinant Alpha 1-Antitrypsin

... has begun to develop products to treat a variety of diseases including hereditary emphysema, COPD, asthma and cystic fibrosis." Joseph McGuirl joined ... AAT is currently marketed by several companies for the treatment of hereditary emphysema and generates over $200 Million in revenues, annually. The ...

Nobel Prize-Winning Discovery

... thereby providing information about their functions. Genes linked with hereditary conditions can be deactivated, or turned "off," in "knock-out mice." ... into uncharted waters of possibility and promise. Pathways of many hereditary conditions -- including obesity, high blood pressure and Parkinson's ...

Interim Phase 2 Data for Exelixis' XL880 Show Anti-Tumor Activity in Papillary Renal Cell Cancer

... open-label phase 2 study is designed to enroll up to 34 patients with hereditary or sporadic papillary renal cell carcinoma. Eligible patients are ... potent activity against mutationally activated forms of MET found in hereditary papillary renal cell carcinomas. The compound also demonstrated ...

Is Your Hair Taking a Break?

... preventing future hair loss." Female-Pattern Hair Loss The most common form of hair loss in women is female-pattern hair loss, which is a hereditary condition also referred to as androgenetic alopecia. While pattern hair loss affects both men and women, it is very different in women and does not ...

SIMPONI(TM) (golimumab) Receives Positive Opinion From CHMP for Once-Monthly Subcutaneous Treatment of Rheumatoid Arthritis, Psoriatic Arthritis and Ankylosing Spondylitis

... rubber containing latex, and may cause allergic reactions in individuals sensitive to latex. SIMPONI also contains sorbitol; patients with rare hereditary problems of fructose intolerance should not take SIMPONI. Women of childbearing potential must use adequate contraception to prevent pregnancy ...

Cystic Fibrosis - Orphan Drug Designation for Innovative Treatment Against Lung Infections by Axentis Pharma AG

... inner cell compartment, which leads to rapid cell death. About cystic fibrosis Cystic fibrosis is the most common life-threatening hereditary disease amongst Caucasian populations. The disease is caused by a mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene ...

Pivotal Clinical Data Published for the Only Laser Phototherapy Device Cleared by the FDA for Hair Growth

... "This is the first controlled clinical trial published that proved the clinical efficacy and safety of a laser phototherapy device for treating hereditary hair loss." Clinical assessment of treated scalp sites was carried out by objective evaluation of macro-images using dot mapping and computer ...

Genetic Source of Rare Childhood Cancer Found; Gene is Implicated in Other Cancers

... those of normal tissue, which pointed toward a possible role for DICER1 in cancer," says senior author Paul Goodfellow, Ph.D., co-director of the hereditary Cancer Core at the Siteman Cancer Center at Washington University School of Medicine and Barnes-Jewish Hospital in St. Louis. "Very recently, other ...

ViroPharma Submits Comments on Vancocin(R) Draft Bioequivalence Guidance

... strains, and Cinryze(TM) (C1 inhibitor (human)) for routine prophylaxis against angioedema attacks in adolescent and adult patients with hereditary angioedema (HAE), also known as C1 inhibitor deficiency (for prescribing information on ViroPharma's commercial products, please download the package ...

ViroPharma Announces Discontinuation of Maribavir Phase 3 Study in Liver Transplant Patients

... strains, and Cinryze(TM) (C1 inhibitor (human)) for routine prophylaxis against angioedema attacks in adolescent and adult patients with hereditary angioedema (HAE), also known as C1 inhibitor deficiency (for prescribing information on ViroPharma's commercial products, please download the package ...

Video: Tasigna(R) Demonstrates Rapid Response as Initial Therapy in Life-Threatening Form of Leukemia

... and 1 hour after taking dose. Lactose Since the capsules contain lactose, Tasigna is not recommended for patients with rare hereditary problems of galactose intolerance, severe lactase deficiency, or of glucose-galactose malabsorption. Pregnancy Fetal harm can ...

More Than 200 Abstracts at ASH and SABCS Reveal Potential Compelling Patient Benefits From Novartis Oncology Current and Pipeline Therapies

... before and 1 hour after taking dose. Lactose Since the capsules contain lactose, Tasigna is not recommended for patients with rare hereditary problems of galactose intolerance, severe lactase deficiency, or of glucose-galactose malabsorption. Pregnancy Fetal harm can occur ...

ASGE and The Pancreatic Cancer Action Network Partner to Raise Awareness About Pancreatic Cancer

... 30 percent are thought to result from cigarette smoking. Some other risk factors scientists are currently studying include: diabetes, chronic and hereditary pancreatitis (inflammation of the pancreas), obesity, physical activity, and diet. "Screening for pancreatic cancer in relatives of affected ...

Raptor Pharmaceuticals to Collaborate with Centre Hospitalier Universitaire d'Angers for Phase II Clinical Trial in Huntington's Disease

... in the clinical trial by determining the BDNF levels in the patients. About Huntington's Disease Huntington's is a rare, progressive, and hereditary neurological disease that often leads to death within 15 to 20 years after diagnosis. The disease is thought to affect as many as 20,000 patients in ...

Young Women Diagnosed with Early-Stage Breast Cancer More Likely to Die than Older Women

... to genetics. Indeed, about 20 percent of young women diagnosed with breast cancer have a significant family history of the disease. Unfortunately, hereditary breast cancers tend to have a poorer outcome than nonhereditary types. Dr. Margenthaler is hopeful that this research will increase the awareness ...

Photos: NIH Launches New Web Site for Parents on Medical Research Studies for Children

... information is key for parents to feel comfortable, adds one parent, whose daughter is participating in a study on a treatment for Marfan syndrome, a hereditary condition that weakens connective tissue, often leading to dangerous problems in the heart and blood vessels, as well as bones and joints, eyes, and ...

Athera Biotechnologies and Dyax Announce a Collaboration to Discover and Develop Antibodies for Cardiovascular Inflammation

... announced the submission of its Biologics License Application with the U.S. Food and Drug Administration for approval of DX-88 for the treatment of hereditary angioedema (HAE). DX-88 has orphan drug designation in the U.S. and E.U., as well as Fast Track designation in the U.S., for the treatment of acute ...

Cystic Fibrosis Axentis Pharma Initiates Clinical Trial for Lung Infections

... Lukacs will contribute to the intellectual capital of Axentis Pharma." About cystic fibrosis Cystic fibrosis is the most common life-threatening hereditary disease amongst Caucasian populations. The disease is caused by a mutation in the CFTR (Cystic Fibrosis Transmembrane Conductance Regulator) gene ...

Boston University and The Center for Human Genetics, Inc. Announce Change in Testing Services

... developmental disorders caused by mutation of the ARX gene, Duchene's muscular dystrophy, myotonic dystrophy (DM1), Freidreich's Ataxia (FRDA), hereditary neuropathy with pressure palsies, polycystic kidney disease, spinal muscular atrophy (SMN1), spinocerebellar ataxia (SCA1, 2, 3, 6, 7 and 8) and ...

Annual FIT Testing Detects Colorectal Cancer Two Years Earlier Than Colonoscopy Alone, Study of High-Risk Population Finds

... a personal history of colorectal cancer who have had a normal 3-year colonoscopy, and more frequent colonoscopies if polyps are detected or a risk of hereditary nonpolyposis colorectal cancer (HNPCC) is suspected. For patients at average risk over the age of 50, ACS guidelines include colonoscopy every ten ...

BiPar Sciences Expands Phase 2 Clinical Trials of BSI-201, a Novel DNA Repair Inhibitor, in Ovarian Cancer

... the first poly ADP-ribose polymerase (PARP) inhibitor in its DNA repair portfolio, as a monotherapy for patients whose ovarian cancer is linked to a hereditary genetic defect. Clinicians at Memorial Sloan-Kettering Cancer Center will enroll patients who have a mutation in their BRCA1 or BRCA2 genes. "We ...

Gene Therapy Improves Vision in Patients with Congenital Retinal Disease

... for the ongoing research for the past 14 years. The F.M. Kirby Center was the first molecular biology center devoted to developing gene therapy for hereditary causes of vision loss. The Center serves as home to the laboratories of seven investigators who conduct research on the cellular and molecular ...

Ranolazine Shortens QT Interval and Improves Cardiac Relaxation in Study of Patients With Genetic Sodium Channel Disorder

... -- CV Therapeutics, Inc. (Nasdaq: CVTX ) announced today that ranolazine significantly (p<0.001) shortened the QT interval of patients with a hereditary form of long QT syndrome called LQT3, which is caused by a genetic mutation in the late sodium channel and can be associated with heart rhythm ...

CSL Behring Submits BLA Requesting Approval of C1-Esterase Inhibitor for the Treatment of Hereditary Angioedema

... therapies for HAE in North America. "Submission of the BLA for C1-INH brings CSL Behring one step closer to providing therapy for hereditary angioedema," said Val Romberg, Senior Vice President of Research and Development at CSL Behring. "We are confident that our expertise in developing ...

Arriva Pharmaceuticals Moves Forward to Phase 2b Development of Lead Inhaled Protein rAAT Replacement Therapy for Hereditary Emphysema

... Nordic Biotech II KS and from our earlier lead investor, MPM Capital, allows us to continue the development of recombinant alpha-one antitrypsin for hereditary emphysema," said Sue Preston, CEO, Arriva Pharmaceuticals. "With a committed, experienced management team, we are now focused on the clinical ...

CSL Behring Reaches Primary Endpoint in Clinical Study of C1-INH for Treatment of Hereditary Angioedema

... that it has reached the primary endpoint of a Phase III clinical trial of human pasteurized C1-inhibitor (C1-INH) concentrate to treat patients with hereditary angioedema (HAE), a rare genetic disorder that can lead to painful and sometimes life-threatening attacks of edema (swelling) of the face, airway, ...

Study Shows Need to Test More COPD and Asthma Patients for Underdiagnosed Pulmonary Disease

... (AAT), suggesting the need for broader criteria for AAT deficiency testing. AAT deficiency, also known as Alpha-1, is a widely undiagnosed hereditary disorder that is usually fatal in its severe form. Alpha-1 is estimated to affect up to 100,000 Americans, but up to 95 percent are undiagnosed or ...

GlaxoSmithKline Accelerates Review of Exelixis' XL880

... studies, XL880 inhibited its targets with nanomolar potency, and retained potent activity against mutationally activated forms of MET found in hereditary papillary renal cell carcinomas. The compound also demonstrated dose-dependent tumor growth inhibition in models of breast cancer, colorectal cancer, ...

Lev Pharmaceuticals Reports Positive Results in Pivotal Phase III Trial for Hereditary Angioedema

... inhibitor (C1-INH) for the acute treatment of hereditary angioedema (HAE). In the acute study, which is part of the company's CHANGE trials (C1-inhibitor in hereditary Angioedema Nanofiltration Generation evaluating ... company's Chief Executive Officer. About hereditary Angioedema HAE, ...

Lev Pharmaceuticals Completes Phase III Prophylaxis Trial of C1-Esterase Inhibitor for Hereditary Angioedema

... sustained relief of acute HAE symptoms. HAE, or hereditary C1-inhibitor deficiency, is a rare ... be used to expand the label indication. About hereditary Angioedema HAE is a genetic disorder ... more information about Lev's studies for hereditary angioedema should contact the Company directly at ...

DX-88 for Hereditary Angioedema Meets Primary and Secondary Endpoints in Phase 3 Trial (EDEMA3)

... trial, EDEMA3, for its lead product candidate DX-88 (ecallantide), for hereditary angioedema (HAE). A webcast presentation of the EDEMA3 data is scheduled ... three Phase 2 trials and a Phase 3 trial of DX-88 for the treatment of hereditary angioedema (HAE). A confirmatory study, known as EDEMA4, is planned and ...

Cogane Data Presented at ‘11th International Congress of Parkinson’s Disease and Disorders’

... The cause of Parkinson's disease in the majority of cases is unknown (idiopathic Parkinson's disease), though some cases have been found to have a hereditary component (familial Parkinson's disease) and possible mechanisms include oxidative damage of nerve cells coupled with loss of neurotrophic factors. ...

Haemacure Announces Preliminary Findings of its Plasma Discard Analysis

... clot buster and offers a natural alternative to synthesized solutions to address an unmet need of the market. A1PI is used by patients suffering from hereditary emphysema resulting from A1PI deficiency. A1PI deficiency affects between 60,000 to 100,000 people in the United States. According to Cowen and ...