Hypophosphatasia

Type: Keyphrase
Name: Hypophosphatasia
First reported Sep 19 2014 - Updated Sep 19 2014 - 3 reports

Paediatric Patients with Hypophosphatasia Receiving Investigational Asfotase Alfa Had Sustained Improvements in Growth and Physical Function

(PR-inside.com) Alexion Pharmaceuticals today announced that researchers presented data from the open-label extension phases of two Phase 2 studies of asfotase alfa, an investigational enzyme replacement therapy, in paediatric patients with hypophosphatasia ... [Published PR inside - Sep 19 2014]
First reported Sep 19 2014 - Updated Sep 19 2014 - 1 reports

Paediatric Patients with Hypophosphatasia -2-

5. Whyte MP, Greenberg CR, Salman N, et al. Enzyme-replacement therapy in life-threatening hypophosphatasia. N Engl J Med. 2012;366(10):904-913.6. Seshia SS, Derbyshire G, Haworth JC, Hoogstraten J. Myopathy with hypophosphatasia. Arch Dis Child. 1990; ... [Published Scottrade - Sep 19 2014]
First reported Sep 14 2014 - Updated Sep 14 2014 - 2 reports

Improved Survival Observed in Pediatric Patients -2-

New data presented at ASBMR showed the following:-- In patients treated with asfotase alfa, a significant and clinicallymeaningful decrease in disability was observed, as measured by the ChildHealth Assessment Questionnaire (CHAQ), from a median of 1 ... [Published Scottrade - Sep 14 2014]
First reported Sep 10 2014 - Updated Sep 10 2014 - 1 reports

Alexion’s Asfotase Alfa For HPP Receives Orphan Status In Japan

By Estel Grace MasangkayAlexion Pharmaceuticals reported that it has received Orphan Drug Designation from the Japanese Ministry of Health, Labor, and Welfare (MHLW) for its asfotase alfa as treatment for patients with hypophosphatasia (HPP).Asfotase ... [Published Bioresearch Online - Sep 10 2014]
First reported Sep 08 2014 - Updated Sep 08 2014 - 1 reports

Asfotase Alfa Granted Orphan Drug Designation in Japan

. (NASDAQ:ALXN) today announced that Japan’s Ministry of Health, Labour and Welfare (MHLW) has granted orphan drug designation (ODD) to asfotase alfa for the treatment of patients with hypophosphatasia (HPP), a genetic, chronic and progressive ultra-rare ... [Published Pettinga Financial Advisors - Sep 08 2014]
First reported Aug 21 2014 - Updated Aug 21 2014 - 1 reports

ALEXION PHARMACEUTICALS : Researchers to Present Asfotase Alfa Data at American Society for Bone and Mineral Research Meeting

Alexion Pharmaceuticals reported that new data from studies of asfotase alfa in pediatric patients with hypophosphatasia will be presented at the American Society for Bone and Mineral Research 2014 Annual Meeting, which takes place Sept. 12-15 , in Houston, ... [Published 4 Traders - Aug 21 2014]
First reported Aug 13 2014 - Updated Aug 13 2014 - 1 reports

Researchers to Present New Data on Asfotase Alfa in Pediatric Patients with Hypophosphatasia at the American Society for Bone and Mineral Research 2014 Annual Meeting

CHESHIRE, Conn.--(BUSINESS WIRE)--Alexion announced that new data from studies of asfotase alfa in pediatric patients with hypophosphatasia will be presented at the American Society for Bone and Mineral Research 2014 Annual Meeting. ... [Published Business Wire Health News - Aug 13 2014]
First reported Jul 24 2014 - Updated Jul 24 2014 - 3 reports

European Medicines Agency Accepts Marketing Authorization Application for Asfotase Alfa as a Treatment for Patients with Hypophosphatasia

CHESHIRE, Conn.--(EON: Enhanced Online News)--Alexion announced that the marketing authorization application for asfotase alfa has been validated and granted accelerated assessment by the European Medicines Agency. ... [Published EON Science - Jul 24 2014]
First reported May 06 2014 - Updated May 06 2014 - 3 reports

New Data Detailing the Devastating Nature of Hypophosphatasia (HPP) and Treatment Effect of Asfotase Alfa in Infants, Young Children and Juveniles with HPP Presented at the Pediatric Academic Societies Meeting

CHESHIRE, Conn.--(EON: Enhanced Online News)--Alexion presented natural history data in patients with severe perinatal & infantile HPP. Early and sustained improvements were observed in infants, children & juveniles receiving asfotase alfa. ... [Published EON Science - May 06 2014]
First reported Apr 11 2014 - Updated Apr 11 2014 - 3 reports

Researchers to Present New Data on Asfotase Alfa in Infants and Juveniles with Hypophosphatasia at the Joint Meeting of the Pediatric Academic Societies and the Asian Society for Pediatric Research

CHESHIRE, Conn.--(EON: Enhanced Online News)--Alexion announced data from two studies examining long-term efficacy and safety of asfotase alfa in infants and juveniles with hypophosphatasia to be presented at the upcoming PAS and ASPR meeting. ... [Published EON Science - Apr 11 2014]

Quotes

...Katherine L Madson, M D , Ph D , from the Center for Metabolic Bone Disease and Molecular Research at Shriners Hospitals for Children, St. Louis. "These children previously had difficulty walking and muscle weakness that limited their physical function, so the improvements seen in the study were meaningful for these patients and their families."
...Madson. "These new physical function data, which include an improved ability to run, jump and hop, reflect better strength and agility for these children who were previously faced with significant skeletal and muscular challenges."
-Abstract FR0435: "Enzyme-Replacement Therapy in Life- Threatening Hypophosphatasia: The 3-Year Experience with Asfotase Alfa" Whyte MP, et al
Alexion Pharmaceuticals CEO Leonard Bell said: "Hypophosphatasia is a devastating disease for patients and their families due to progressive deterioration of bones and muscle weakness, which can result in impaired respiratory function, severe disability and death."

More Content

All (20) | News (7) | Reports (0) | Blogs (12) | Audio/Video (0) | Fact Sheets (0) | Press Releases (0)
sort by: Date | Relevance
Paediatric Patients with Hypophosphatasia Recei... [Published PR inside - Sep 19 2014]
Paediatric Patients with Hypophosphatasia Recei... [Published Scottrade - Sep 19 2014]
Paediatric Patients with Hypophosphatasia -2- [Published Scottrade - Sep 19 2014]
Paediatric Patients with Hypophosphatasia Recei... [Published Business Wire Health News - Sep 19 2014]
Improved Survival Observed in Pediatric Patient... [Published Scottrade - Sep 14 2014]
Improved Survival Observed in Pediatric Patient... [Published Business Wire Health News - Sep 14 2014]
Alexion’s Asfotase Alfa For HPP Receives Orphan... [Published Bioresearch Online - Sep 10 2014]
Asfotase Alfa Granted Orphan Drug Designation i... [Published Pettinga Financial Advisors - Sep 08 2014]
ALEXION PHARMACEUTICALS : Researchers to Presen... [Published 4 Traders - Aug 21 2014]
Researchers to Present New Data on Asfotase Alf... [Published Business Wire Health News - Aug 13 2014]
EMA accepts Alexion’s marketing authorisation a... [Published Pharmaceutical Technology - Jul 27 2014]
European Medicines Agency Accepts Marketing Aut... [Published EON Science - Jul 24 2014]
European Medicines Agency Accepts Marketing Aut... [Published Business Wire Science: Science News - Jul 24 2014]
European Medicines Agency Accepts Marketing Aut... [Published Business Wire Health News - Jul 24 2014]
New Data Detailing the Devastating Nature of Hy... [Published EON Science - May 06 2014]
New Data Detailing the Devastating Nature of Hy... [Published Business Wire Science: Science News - May 06 2014]
New Data Detailing the Devastating Nature of Hy... [Published Business Wire Health News - May 06 2014]
Researchers to Present New Data on Asfotase Alf... [Published EON Science - Apr 11 2014]
Researchers to Present New Data on Asfotase Alf... [Published Business Wire Science: Science News - Apr 11 2014]
Researchers to Present New Data on Asfotase Alf... [Published Business Wire Health News - Apr 11 2014]
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Paediatric Patients with Hypophosphatasia Recei... [Published Business Wire Health News - Sep 19 2014]
DUBLIN, Ireland--(BUSINESS WIRE)--Alexion announced that researchers presented data from the open-label extension phases of two Phase 2 studies of asfotase alfa in paediatric patients with hypophosphatasia at the 53rd ESPE Meeting. ...
Improved Survival Observed in Pediatric Patient... [Published Business Wire Health News - Sep 14 2014]
CHESHIRE, Conn.--(BUSINESS WIRE)--Alexion announced data from an integrated analysis of survival from two open-label, Phase 2 studies of asfotase alfa in pediatric patients with hypophosphatasia compared with a natural history study. ...
Researchers to Present New Data on Asfotase Alf... [Published Business Wire Health News - Aug 13 2014]
CHESHIRE, Conn.--(BUSINESS WIRE)--Alexion announced that new data from studies of asfotase alfa in pediatric patients with hypophosphatasia will be presented at the American Society for Bone and Mineral Research 2014 Annual Meeting. ...
European Medicines Agency Accepts Marketing Aut... [Published EON Science - Jul 24 2014]
CHESHIRE, Conn.--(EON: Enhanced Online News)--Alexion announced that the marketing authorization application for asfotase alfa has been validated and granted accelerated assessment by the European Medicines Agency. ...
European Medicines Agency Accepts Marketing Aut... [Published Business Wire Science: Science News - Jul 24 2014]
CHESHIRE, Conn.--(BUSINESS WIRE)--Alexion announced that the marketing authorization application for asfotase alfa has been validated and granted accelerated assessment by the European Medicines Agency. ...
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