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Fabry in Medical News

Preliminary Results from Phase 2 Genzyme Study Highlight Potential of Novel Oral Compound for Gaucher Disease

...gram that involved more than 120 subjects in three separate studies. In addition to Gaucher disease, there are a variety of other conditions including fabry disease that can be caused by malfunctions in the pathway targeted by this molecule. The compound is exclusively licensed from the University of Michi...

VIDEO from Medialink and Shire plc: Rare Diseases Must Be Top of the Health Agenda

...solation for the patient's family. Case study: fabry Disease (also known as Anderson-Fabry Disease) is ...of the symptoms and the slow progressive course of fabry disease, there is an average delay between the ons...uced by 20 years in men and 15 years in women with fabry disease, compared with the general population. ...

Genzyme Delivers Strong Fourth Quarter to Conclude Outstanding Year

...mpared with $424 million in 2007. The European Commission has granted full marketing authorization for Fabrazyme, making the product the only fabry disease treatment to earn this designation in Europe. -- Sales of Cerezyme(R) (imiglucerase for injection) are expected to reach $1.22-$1.24...

Further Upgrade to 2007 Financial Guidance Based on Strong Revenue Growth; VYVANSE Demand on Track

...'s existing markets, with exchange rate movements against the US dollar accounting for the remaining 9% increase. Human Genetic Therapies REPLAGAL - fabry disease Sales for the three months to September 30, 2007 were $40.7 million, an increase of 26% compared to the same period in 2006 (2006: $32.4 mill...

New DNA Tests Advances Treatment of Lysosomal Storage Diseases

...the first genetic disorder that could be treated effectively with enzyme replacement therapy (ERT). Over the past decade, ERT has become available for fabry disease, Mucopolysaccharidosis Type VI (MPS VI - Maroteaux-Lamy), and MPS I (Hurler, Hurler-Scheie, or Scheie syndrome). ERT also is in development fo...

Rare Genetic Disorder Could Cause Strokes In Young People

...eatment, says a study. The disorder known as fabry disease is characterised by attacks of burning pai...m unexplained strokes to find out whether they had fabry disease. They found that four percent of the...eople, aged 18-55 years who had a stroke, also had fabry disease. The strokes occurred about a decade earli...
Fabry in Medical Technology

Amicus Therapeutics Commences Phase 3 Trial Evaluating Amigal(TM) for the Treatment of Fabry Disease

- FDA Agreement Reached on Phase 3 Study Design, Primary Endpoint and Histological Methodology - CRANBURY, N.J., June 22 /PRNewswire-FirstCall/ -- Amicus Therapeutics (Nasdaq: FOLD ) today announced it has commenced the U.S. registration Phase 3 trial with its investigational drug, Amigal...

Amicus Therapeutics Announces Successful Completion of End of Phase 2 Meeting With FDA for Amigal in Fabry Disease

Agency Supports Amigal Move to Phase 3; Eligible for Accelerated Approval; Final Global Regulatory Plan Expected by Year End CRANBURY, N.J., Aug. 4 /PRNewswire-FirstCall/ -- Amicus Therapeutics (Nasdaq: FOLD ), a biopharmaceutical company developing small molecule, orally-administere...

Amicus Therapeutics Presents Data From Preclinical and Phase 1 Studies of Amigal for Fabry Disease

Studies in Mice Show Reduction of Enzyme Substrate CRANBURY, N.J., March 19, 2007 /PRNewswire/ -- Amicus Therapeutics, a biopharmaceutical company developing small-molecule, orally administered pharmacological chaperones for the treatment of human genetic diseases, announced today that the Company...

Amicus Therapeutics Suspends Enrollment for Phase 2 Clinical Trial of AT2220 for Pompe Disease

...es with its investigational drugs Amigal(TM) (migalastat hydrochloride) for fabry disease and Plicera(TM) (afegostat tartrate) for Gaucher disease. Ab...Amicus has completed Phase 2 clinical trials of Amigal for the treatment of fabry disease and is conducting Phase 2 clinical trials of Plicera for the treatm...

Amicus Therapeutics Announces Positive Progress of Three Lead Clinical Programs and General Outlook for 2009

...al therapeutic drug candidate for the treatment of fabry disease. Amicus, along with its partner, Shire Hu...Phase 3 development of Amigal for the treatment of fabry disease in the second quarter of 2009. Amicus also...al (migalastat hydrochloride) for the treatment of fabry Disease Amicus previously announced in Augus...

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

... of the Web site videos, a young teen enrolled in a study of treatments for fabry disease says that being in a research study is "going to be worth it in the...helping myself and future generations and people who have the disease now." fabry disease is an inherited condition that can cause severe pain, vision proble...

Amicus Therapeutics Announces Second Quarter 2008 Financial Results

...essful End of Phase 2 Meeting with the FDA for the fabry Program and Initiation of Phase 2 Clinical Trial ...ll be focused on finalizing the regulatory plan in fabry as well as executing on our Gaucher and Pompe Phas...pated 2008 cash burn." Program Advancements fabry Disease: Amigal(TM) (migalastat hydrochloride) i...

Amicus Therapeutics Begins Phase 2 Clinical Trial of AT2220 in Pompe Disease

..., which are severe, chronic genetic diseases with unmet medical needs. Amicus has completed Phase 2 clinical trials of Amigal(TM) for the treatment of fabry disease and is conducting Phase 2 clinical trials of Plicera(TM) for the treatment of Gaucher disease. Forward-Looking Statements Amicus cautions ...

Amicus Therapeutics Presents Data from Clinical Ex Vivo Response Study and Phase 1 Studies of AT2220

..., which are severe, chronic genetic diseases with unmet medical needs. Amicus has completed Phase 2 clinical trials of Amigal(TM) for the treatment of fabry disease and is conducting Phase 2 clinical trials of Plicera(TM) for the treatment of Gaucher disease. The Company recently completed Phase I clinical...

Amicus Therapeutics to Present at the 26th Annual JPMorgan Healthcare Conference

...us has completed Phase 2 clinical trials of Amigal(TM) for the treatment of fabry disease and is conducting Phase 2 clinical trials of Plicera(TM) for the tr...he completion of the Phase 2 clinical trial for Amigal for the treatment of fabry disease, the plans for the Phase 3 clinical trial for Amigal, the Phase II ...
Fabry in Biological News

Undergraduates develop 'dirt-powered' microbial fuel cells to light Africa

... undergraduate students Stephen Lwendo 10 (computer science and engineering) and David Sengeh 10 (bioengineering), who are both from Africa, Alexander fabry 09 (history of science and physics), alumnae Zo Sachs-Arellano 07 (a philosophy concentrator who co-founded the Namibia Connection Youth Network), and...

Report warns about carbon dioxide threats to marine life

...imals is likely to impact marine food webs and has the potential to substantially alter the biodiversity and productivity of the ocean," says Victoria fabry of California State University, San Marcos, who is another of the report's authors. Threats to major ecosystems Several other major ecosystems...
Fabry in Biological Technology

Amicus Therapeutics Presents Positive Results From Phase 2 Extension Study of Amigal(TM) for Fabry Disease at ACMG 2009 Annual Meeting

Data provide support for expected Phase 3 program CRANBURY, N.J., March 28 /PRNewswire-FirstCall/ -- Amicus Therapeutics (Nasdaq: FOLD ) announced today positive results from its ongoing Phase 2 extension study of its investigational drug, Amigal(TM) (migalastat HCL) for Fabry disease. The...

Amicus Therapeutics Announces Positive Results From Phase 2 Clinical Trials of Amigal(TM) for Fabry Disease

CRANBURY, N.J., Dec. 19 /PRNewswire-FirstCall/ -- Amicus Therapeutics Inc. (Nasdaq: FOLD ), a biopharmaceutical company developing small-molecule, orally administered pharmacological chaperones for the treatment of human genetic diseases, announced today positive results from its recently com...

Amicus Therapeutics Announces Second Quarter 2009 Financial Results

...ement for Amicus with our lead program, Amigal for fabry disease, entering Phase 3 development." Crowley c...rtant, novel treatment to patients who suffer with fabry disease." Clinical Program Updates ... (migalastat hydrochloride) for the treatment of fabry disease In the second quarter of 2009, Ami...

Shire plc: Core Portfolio of Products Delivers 20% Sales Growth

...primarily sold in US dollars and Euros). The sales growth was driven by increased volumes across all regions where ELAPRASE is sold. REPLAGAL - fabry disease Product sales for the three months to June 30, 2009 were $44.4 million, a decrease of 1% (2008: $44.7 million). Expressed on a CER basi...

Shire begins the year with a strong performance

...rily sold in US dollars and Euros). The sales growth was driven by increased unit sales in Europe, North America, and Latin America. REPLAGAL - fabry disease Product sales for the three months to March 31, 2009 were $40.2 million, a decrease of 5% compared to the same period in 2008 (2008: $4...

Publication of Shire plc's Annual Report 2008

...LICERA in markets outside the US. AMIGAL (HGT-3310 for the treatment fabry disease) AMIGAL is an orally-administered, small molecule pharmacological chaperone being developed for the treatment of fabry disease. AMIGAL has received orphan drug designation by the EMEA, which may...

Shire Delivers Excellent Growth for the Year, With the new Product Portfolio Achieving Sales of $1 Billion

... ELAPRASE is sold: Europe, North America, Latin America and Asia Pacific. On a constant exchange rate basis, sales increased by 64%. REPLAGAL - fabry disease Sales for the year to December 31, 2008 were $176.1 million, an increase of 22% compared to the same period in 2007 (2007: $143.9 milli...

Amicus Therapeutics Announces Change to Board of Directors

...ders, which are severe, chronic genetic diseases with unmet medical needs. Amicus has completed Phase 2 clinical trials of Amigal for the treatment of fabry disease and is conducting Phase 2 clinical trials of Plicera for the treatment of Gaucher disease and AT2220 for the treatment of Pompe disease. ...

Amicus Therapeutics Announces Fourth Quarter and Full Year 2008 Financial Results

...M) (migalastat hydrochloride) for the treatment of fabry disease In the fourth quarter of 2008, Amicu...Phase 3 development of Amigal for the treatment of fabry disease in the second quarter of 2009. In Jan...-3, the substrate that accumulates in the cells of fabry patients. Amicus is continuing discussions with th...

Amicus Therapeutics Announces Fourth Quarter and Year End 2008 Financial Results Release Date

...ders, which are severe, chronic genetic diseases with unmet medical needs. Amicus has completed Phase 2 clinical trials of Amigal for the treatment of fabry disease and is conducting Phase 2 clinical trials of Plicera for the treatment of Gaucher disease and AT2220 for the treatment of Pompe disease. ...
Fabry in Biological Definition

X chromosome

...he X chromosome: Alport syndrome androgen insensitivity syndrome Charcot-Marie-Tooth disease Coffin-Lowry syndrome fabry disease fragile X syndrome hemophilia incontinentia pigmenti Lesch-Nyhan syndrome Menkes syndrome Duchenne and Be...
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