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Advances in Testing and Tx Improve Fabry Disease Pts Lives

Earlier diagnostic screening, routine and emerging therapies, and increased awareness are helping people with the lysosomal storage disorder Fabry disease lead a longer, healthier life. ....

United States , Chelseaj Lauderdale , Lizbeth Mellin , Monroe Carell Jr , Sanjay Rastogi , Gerald Vincent Raymond , Los Angeles , Genzyme Sanofi , Drug Administration , Fabry Disease Program , Amicus Therapeutics , Science Writer , Medscape Professional Network , Idorsia Pharmaceuticals Ltd , Fabry Outcome Survey , Protalix Biotherapeutics , University Of Florida Health Jacksonville , Four Fabry , Neurology Reviews , Neurological Disease Special , Lysosomal Storage Disease Center , Johns Hopkins Medicine , Florida Health , Disease Program , Fabry Disease Clinic , Chiesi Global Rare Diseases ,

FDA OKs New Drug for Fabry Disease

Pegunigalsidase alfa (Elfabrio) is an intravenously administered enzyme replacement therapy to treat adults with confirmed Fabry disease. ....

Protalix Biotherapeutics , Sanofi Genzyme , Dror Bashan , Drug Administration , Chiesi Global Rare , Lysosomal Storage Disease , Renal Failure , Enal Insufficiency , Idney Insufficiency , Kidney Failure , Drug Development , Membranoproliferative Glomerulonephritis , Clinical Research , Clinical Trials , Linical Studies , Angiokeratoma Corporis Diffusum , Mabry Syndrome , Fabry Disease , Anderson Fabry Disease , Eramide Trihexosidosis , Weeley Klionsky Disease , Blood Vessels ,

My baby will never walk or talk after being born with rare brain disease

Lucy Landman, now one, from California, has been diagnosed with a rare brain condition that pushes her development off a cliff . Parents are currently fundraising to develop a treatment. ....

United States , Lucy Landman , Rare Diseases Clinical Research Network , Mabry Syndrome ,

Small Fiber Neuropathy Rising in the US, But Why Is a Mystery

Over the past two decades there has been a significant increase in the number of adults in the United States with small fiber neuropathy, but in many cases, no cause can be determined. ....

United States , Mayo Clinic In Rochester , Ann Arbor , Salt Lake City , University Of Utah , J Robinson Singleton , Brian Callaghan , Christopherj Klein , Important Research , University Of Michigan , Mayo Clinic , Medscape Medical , Diabetic Neuropathy , Diabetic Neuropathies , Iabetic Nerve Damage , Diabetic Foot , Diabetes Mellitus , Peripheral Neuropathy , Erectile Dysfunction , Body Mass Index Bmi , Heart Failure , Myocardial Infarction , Sjogrens Syndrome , Sjogren Syndrome , Angiokeratoma Corporis Diffusum , Mabry Syndrome ,