Fabry Disease News and Research

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Fabry disease (also known as Fabry's disease, Anderson-Fabry disease, angiokeratoma corporis diffusum and alpha-galactosidase A deficiency) is a rare X-linked recessive (inherited) lysosomal storage disease, which can cause a wide range of systemic symptoms.

GSK, Angiochem to develop and commercialize LSD treatments

Angiochem announced today a global collaboration with GlaxoSmithKline (GSK) to discover, develop and commercialize treatments for lysosomal storage diseases.

27 Feb 2012

Researchers discover key interaction at heart of promising new treatment for Fabry disease

A research team led by biochemist Scott Garman at the University of Massachusetts Amherst has discovered a key interaction at the heart of a promising new treatment for a rare childhood metabolic disorder known as Fabry disease.

23 Dec 2011

PDUFA goal date of NDA for Protalix's taliglucerase alfa extended

Protalix BioTherapeutics, Inc., announced today that it received notification from the U.S. Food and Drug Administration (FDA) that the FDA has extended the Prescription Drug User Fee Act (PDUFA) goal date of the New Drug Application (NDA) for taliglucerase alfa to May 1, 2012, a three-month extension from the previous PDUFA date of February 1, 2012.

6 Dec 2011

iBio receives U.S. patent entitled 'System for Expression of Genes in Plants'

iBio, Inc. today announced notice of issuance from the United States Patent and Trademark Office for US Patent 8,058,511, entitled "System for Expression of Genes in Plants," that further extends iBio's patent coverage of viral vectors comprising its iBioLaunch platform.

1 Nov 2011

FDA accepts Protalix taliglucerase alfa NDA resubmission for review

Protalix BioTherapeutics, Inc., announced today that the U.S. Food & Drug Administration (FDA) has accepted for review the resubmission of the taliglucerase alfa New Drug Application (NDA) following the Company's receipt of a Complete Response Letter (CRL) in February 2011.

17 Aug 2011

Spanish patients with Fabry disease show classic observable traits different from rest of Europe

Spanish patients with Fabry disease, a rare hereditary condition where abnormal fatty deposits collect in blood vessels and organs throughout the body, appear to react differently to those in other European countries, according to a study in the August issue of IJCP, the International Journal of Clinical Practice.

20 Jul 2011

EMA approves Shire's new REPLAGAL manufacturing facility in Lexington

Shire plc, the global specialty biopharmaceutical company, today announced that the European Medicines Agency has approved the purification of REPLAGAL® (agalsidase alfa) drug substance at its new manufacturing facility in Lexington, MA, US. REPLAGAL, Shire's enzyme replacement therapy for the treatment of Fabry disease is the first product that will be made available to patients from the new facility.

24 Jun 2011

Shire's Replagal and VPRIV data on Gaucher and Fabry patients presented at ACMG meeting

Shire plc, the global specialty biopharmaceutical company, presented new data for Replagal in patients with Fabry disease, and VPRIV in patients with type 1 Gaucher disease, at the 2011 American College of Medical Genetics' Annual Clinical Genetics Meeting, held in Vancouver, BC, from March 16-20, 2011.

22 Mar 2011

FDA removes clinical hold for Amicus' AT2220 IND for treatment of Pompe disease

Amicus Therapeutics today announced that the U.S. Food and Drug Administration (FDA) has removed the clinical hold for the AT2220 (1-deoxynojirimycin HCI) Investigational New Drug Application (IND). AT2220 is a pharmacological chaperone in development as a treatment for Pompe disease.

9 Mar 2011

Protalix receives FDA CRL for taliglucerase alfa NDA for Gaucher disease

Protalix BioTherapeutics, Inc., announced today that the U.S. Food and Drug Administration (FDA) issued a Complete Response Letter (CRL) regarding the Company's New Drug Application (NDA) for taliglucerase alfa for the treatment of Gaucher disease. Taliglucerase alfa is a plant-cell expressed form of glucocerebrosidase (GCD).

25 Feb 2011

Ibio acquires Orphan Drug Designation and Fabry disease therapeutic program from Kentucky

iBio, Inc. today announced it has acquired Orphan Drug Designation for plant-produced human alpha galactosidase A and related property rights from an affiliate of Kentucky Bioprocessing LLC and has initiated a program, based on its iBioLaunch platform, to develop an improved version of the enzyme for therapy of Fabry disease.

23 Feb 2011

Protalix: New data from taliglucerase alfa, oral GCD trials to be presented at Lysosomal Disease Network Symposium

Protalix BioTherapeutics, Inc., announced today that new clinical data from the switchover trial of taliglucerase alfa in Gaucher disease patients and preclinical data on oral enzyme glucocerebrosidase (oral GCD) will be presented today at the 7th Annual Meeting of the Lysosomal Disease Network: WORLD Symposium 2011 in Las Vegas, Nevada.

19 Feb 2011

Amicus to present additional positive data from Amigal Phase 2 extension study for Fabry disease

Amicus Therapeutics today announced that additional positive data from the ongoing Phase 2 extension study of its investigational drug Amigal™ (migalastat HCl) for Fabry disease will be presented at the Lysosomal Disease Network WORLD Symposium in Las Vegas, Nevada, February 16-18th, 2011.

17 Feb 2011

2010 Mid-Atlantic Bio a great kick-off for hosting 2011 BIO International Convention

The 2010 Mid-Atlantic Bio today completed another successful conference hosting 728 registrants from 22 states and 4 countries, with more than 400 companies and 50 qualified investors participating. Dedicated to promoting the biotechnology industry in the Mid-Atlantic region, the conference is hosted collectively by the region's most influential bioscience and investor associations, the Mid-Atlantic Venture Association (MAVA), the Tech Council of Maryland/MdBio (MdBio) and the Virginia Biotechnology Association (VaBIO).

30 Oct 2010

GSK and Amicus announce Amigal development and commercialization agreement

GlaxoSmithKline PLC (GSK) and Amicus Therapeutics today announced a definitive agreement to develop and commercialize Amigal™ (migalastat HCl), currently in Phase 3 for the treatment of Fabry disease, a rare inherited disorder. Under the terms of the agreement, GSK will receive an exclusive worldwide license to develop, manufacture and commercialize migalastat HCl.

29 Oct 2010

Neuraltus achieves last patient out for NP001, NP002 studies

Neuraltus Pharmaceuticals, a privately held biopharmaceutical company dedicated to developing and commercializing high-impact therapeutics that address critical unmet needs, primarily in the treatment of neurodegenerative diseases, announced today the Company has achieved last patient out in two studies: a Phase 1 clinical study of NP001 in patients with Amyotrophic Lateral Sclerosis (ALS, or Lou Gehrig's disease) and a Phase 1/2 clinical study of NP002 in patients with L-dopa-induced dyskinesias (muscle movement disorders).

5 Oct 2010

Genzyme responds to Sanofi-Aventis' unsolicited acquisition proposal

Genzyme Corp. today confirmed that it has received an unsolicited, non-binding proposal from Sanofi-Aventis to acquire all the outstanding shares of Genzyme for $69 per share in cash. The Genzyme board of directors met last night, unanimously affirmed its previous rejection of Sanofi's proposal, and instructed the company to send Sanofi the following response letter:

30 Aug 2010

Genzyme signs consent decree of permanent injunction, agrees to disgorgement

Genzyme Corp. has signed a consent decree agreeing to correct manufacturing quality violations at its Allston, Mass., manufacturing facility and will turn over to the federal government $175 million in unlawful profits from the sale of products that were made at the plant, the U.S. Food and Drug Administration announced today.

25 May 2010

ADDF awards $210,300 to Amicus Therapeutics to evaluate PCs for treating Alzheimer's disease

The Alzheimer's Drug Discovery Foundation announced today that it has awarded a grant of $210,300 to Amicus Therapeutics (Cranbury, NJ) to evaluate small-molecule, orally delivered pharmacological chaperone compounds as a novel approach for treating Alzheimer's disease.

7 May 2010

Determining personal risk for genetic disorders essential

Genetic diseases affect an estimated 12 million Americans, yet according to a survey of 1,000 people conducted by the Genetic Disease Foundation (GDF), while two-thirds of those surveyed were willing to and saw the benefits of undergoing genetic testing, close to 80 percent had never talked to their physician about genetic screening – an inconsistency that can have serious implications on a person's overall health and the health of their immediate family members.

1 Mar 2010