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Enzyme therapy may prevent skeletal abnormalities associated with neurofibromatosis type-1

Enzyme therapy may prevent skeletal abnormalities associated with neurofibromatosis type-1

An enzyme therapy may prevent skeletal abnormalities associated with the genetic disorder neurofibromatosis type-1, Vanderbilt investigators have discovered. [More]
Alexion Pharmaceuticals reports full year 2013 GAAP and non-GAAP financial results

Alexion Pharmaceuticals reports full year 2013 GAAP and non-GAAP financial results

Alexion Pharmaceuticals, Inc. today announced financial results for the quarter and year ended December 31, 2013. For the three months ended December 31, 2013, Alexion Pharmaceuticals, Inc. ("Alexion" or the "Company") reported net product sales of Soliris® (eculizumab) of $441.9 million, compared to $320.5 million for the same period in 2012. [More]
Asfotase alfa gets Breakthrough Therapy designation from FDA for treatment of patients with HPP

Asfotase alfa gets Breakthrough Therapy designation from FDA for treatment of patients with HPP

Alexion Pharma International Sàrl, a subsidiary of Alexion Pharmaceuticals, Inc., today announced that the U.S. Food and Drug Administration has granted Breakthrough Therapy designation to asfotase alfa for the treatment of patients with hypophosphatasia (HPP) whose first signs or symptoms occurred prior to 18 years of age, including perinatal-, infantile-, and juvenile-onset forms of the disease. [More]
Osteopontin plays a role in X-linked hypophosphatemia

Osteopontin plays a role in X-linked hypophosphatemia

Diagnosed in toddlers, X-linked hypophosphatemia (XLH) is the most common form of heritable rickets, in which soft bones bend and deform, and tooth abscesses develop because infections penetrate soft teeth that are not properly calcified. Researchers at McGill University and the Federal University of Sao Paulo have identified that osteopontin, a major bone and tooth substrate protein, plays a role in XLH. Their discovery may pave the way to effectively treating this rare disease. [More]
Alexion second quarter net product sales increase to $274.7 million

Alexion second quarter net product sales increase to $274.7 million

Alexion Pharmaceuticals, Inc. today announced financial results for the three and six months ended June 30, 2012. [More]
Doctors identify promising new treatment for hypophosphatasia

Doctors identify promising new treatment for hypophosphatasia

Doctors at Washington University School of Medicine in St. Louis, working with Shriners Hospital for Children and other institutions, have identified a promising new treatment for a rare and sometimes life-threatening bone disorder that can affect infants and young children. [More]
Alexion to acquire 100% of capital stock of Enobia

Alexion to acquire 100% of capital stock of Enobia

Alexion Pharmaceuticals, Inc. and Enobia Pharma Corp. today announced that the companies have signed a definitive agreement under which Alexion will acquire 100% of the capital stock of Enobia. [More]

Enobia raises US$40M through private placement

Enobia Pharma Inc., a clinical stage biotech company focused on developing novel therapeutics for serious bone disorders, today announced that it has raised US$40 million through the private placement of approximately 13,724,000 shares of its common stock to new pharmaceutical and financial investors. [More]
Enobia completes patient enrollment in ENB-0040 Phase II study for HPP

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. [More]
Enobia Pharma completes ENB-0040 Phase II study in juveniles with hypophosphatasia

Enobia Pharma completes ENB-0040 Phase II study in juveniles with hypophosphatasia

Enobia Pharma, today announced that it has successfully completed a Phase II study of ENB-0040, a bone targeted enzyme replacement therapy being investigated in juveniles with hypophosphatasia, a serious, rare metabolic bone disorder. [More]
Enobia announces positive results from ENB-0040 Phase II juvenile clinical trial for hypophosphatasia

Enobia announces positive results from ENB-0040 Phase II juvenile clinical trial for hypophosphatasia

Enobia Pharma today announced positive results for its Phase II juvenile clinical trial of ENB-0040 (asfotase alfa), an experimental bone-targeted enzyme replacement therapy intended for the treatment of hypophosphatasia (HPP). Hypophosphatasia is a rare, inherited, and sometimes fatal metabolic bone disease that affects individuals of all ages. [More]
Update on Enobia Pharma's effort to develop ENB-0040 for treating HPP patients

Update on Enobia Pharma's effort to develop ENB-0040 for treating HPP patients

Enobia Pharma today provided an update on key developments in its comprehensive effort to develop ENB-0040 (asfotase alfa), an experimental bone-targeted enzyme replacement therapy, for the treatment of hypophosphatasia. Hypophosphatasia is a rare, inherited, and sometimes fatal metabolic bone disease that affects individuals of all ages. [More]
Enobia reports positive interim data from ENB-0040 enzyme replacement therapy study for children with HPP

Enobia reports positive interim data from ENB-0040 enzyme replacement therapy study for children with HPP

Enobia Pharma today announced positive interim data from a clinical study of ENB-0040, a bone targeted enzyme replacement therapy, under investigation for the treatment of hypophosphatasia (HPP). After 12 weeks of treatment with ENB-0040, children with HPP showed marked improvements in bone mineralization and function including increases in strength, endurance and mobility and reduction in pain. These findings were presented by Dr. Michael Whyte at ENDO 2010, the 92nd Annual Meeting of The Endocrine Society. [More]
Enobia Pharma unveils findings from first HPP self-reported patient survey at ACMG 2010

Enobia Pharma unveils findings from first HPP self-reported patient survey at ACMG 2010

Enobia Pharma Inc. unveiled findings from the first hypophosphatasia self-reported patient survey intended to evaluate the burden of illness associated with HPP at the 2010 American College of Medical Genetics Annual Clinical Genetics Meeting. [More]
McGill, Enobia Pharma collaborate to develop treatments for serious genetic bone diseases

McGill, Enobia Pharma collaborate to develop treatments for serious genetic bone diseases

Dr. Marc McKee, of McGill's Faculty of Dentistry and the Department of Anatomy and Cell Biology, is collaborating closely with Enobia Pharma Inc, a Quebec biotech company, to develop innovative treatments for serious genetic bone diseases. McKee's research looks into the reasons why calcium-phosphate mineral fails to crystallize properly to form strong bones and teeth. [More]
Enobia Pharma initiates study of bone-targeted enzyme replacement therapy for hypophosphatasia

Enobia Pharma initiates study of bone-targeted enzyme replacement therapy for hypophosphatasia

Enobia Pharma announced today the initiation of a Phase 2 clinical study of ENB-0040, a bone-targeted enzyme replacement therapy, under investigation for the treatment of hypophosphatasia (HPP). The six-month study will assess the safety, efficacy and pharmacokinetics of ENB-0040 in up to 12 children with HPP. [More]

Enobia Pharma's clinical study of ENB-0040 for treating hypophosphatasia provides positive results

Enobia Pharma today announced positive data from a clinical study of ENB-0040, a bone targeted enzyme replacement therapy, under investigation for the treatment of hypophosphatasia (HPP). After six months of treatment with ENB-0040, four of five severely affected patients showed marked improvements in bone mineralization, correction of skeletal defects, better respiratory function, including weaning from assisted ventilation, and cognitive and motor development. [More]
Potential hypophosphatasia therapy discovered

Potential hypophosphatasia therapy discovered

Researchers at the Burnham Institute for Medical Research, led by José Luis Millán, Ph.D., have demonstrated in mice the first successful use of enzyme replacement therapy to prevent hypophosphatasia (HPP), a primary skeletal disease of genetic origin. This discovery lays the foundation for future clinical trials for HPP patients. [More]