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Latest Lipid storage disorders Stories

2014-07-27 23:01:10

LifeScienceIndustryResearch.com adds “Metachromatic Leukodystrophy (MLD) Global Clinical Trials Review, H2, 2014” to its store. This report provides elemental information and data relating to the clinical trials on MLD. Dallas, TX (PRWEB) July 27, 2014 Metachromatic leukodystrophy (MLD, also called Arylsulfatase A deficiency) is a lysosomal storage disease which is commonly listed in the family of leukodystrophies as well as among the sphingolipidoses as it affects the metabolism of...

2014-07-23 23:18:14

Investment builds company strength in preparation for clinical trials. Alachua, Fla. (PRWEB) July 22, 2014 CTD Holdings, Inc. (OTCQB:CTDH), a manufacturer and distributor of cyclodextrins for the pharmaceutical, medical device, cosmetics, and other markets, announced today that it has closed on a Private Placement with a group of qualified private investors led by Novit L.P., an investment arm of U.S. Pharmacia. The transaction involved the signing of a Securities Purchase Agreement...

2014-07-22 08:32:03

- Neurotrope enters into world-wide, exclusive license agreement with Mount Sinai; Files related provisional patent for Bryostatin-1 - PLANTATION, Fla., July 22, 2014 /PRNewswire/ -- Neurotrope Bioscience, Inc., a subsidiary of Neurotrope, Inc. (OTCQB: NTRP) has signed a world-wide, exclusive license agreement with the Icahn School of Medicine at Mount Sinai (Mount Sinai) to utilize its proprietary information and data package for the use of Bryostatin-1 in the treatment of...

2014-07-09 23:04:12

Dr. Jeffrey Tate promoted to CEO; C.E. Rick Strattan to serve as Executive Chairman. Alachua, FL (PRWEB) July 09, 2014 CTD Holdings, Inc. (OTCQB:CTDH), a distributor and manufacturer of cyclodextrin products including the orphan drug designated Trappsol(R) Cyclo(TM), today announced the promotion of Dr. Jeffrey Tate to Chief Executive Officer, following the retirement of C.E. Rick Strattan. Dr. Tate will hold the titles of President and CEO of CTD Holdings, Inc.; Mr. Strattan will become...

2014-06-30 16:26:45

Phase 3 Study Meets Primary Endpoint and Six Secondary Endpoints Across Multiple Disease-Related Abnormalities LEXINGTON, Mass., June 30, 2014 /PRNewswire/ -- Synageva BioPharma Corp. (Synageva) (NASDAQ: GEVA), a biopharmaceutical company developing therapeutic products for rare diseases, announced today that the global, randomized, double-blind, placebo-controlled Phase 3 ARISE trial of sebelipase alfa in 66 children and adults with lysosomal acid lipase deficiency (LAL...

2014-06-03 08:31:40

LEXINGTON, Mass., June 3, 2014 /PRNewswire/ -- Synageva BioPharma Corp. (Synageva) (NASDAQ: GEVA), a biopharmaceutical company developing therapeutic products for rare diseases, today announced data presentations and a company-sponsored satellite symposium at the European Atherosclerosis Society (EAS) Congress being held May 31-June 3, 2014 in Madrid, Spain. Logo - http://photos.prnewswire.com/prnh/20140522/90728 Sebelipase alfa continues to improve disease-related abnormalities...

2014-05-07 23:01:17

With expanded screening test, 50 percent of Ashkenazi Jewish individuals are expected to be identified as carriers of at least one of the 38 diseases New York, NY (PRWEB) May 07, 2014 The Icahn School of Medicine at Mount Sinai today announced the launch of its Expanded Carrier Screening Panel for people of Ashkenazi Jewish descent. This test increases the number of diseases covered from 20 to 38, giving Ashkenazi Jewish individuals a 1 in 2 chance of being a carrier for at least one of...

2014-01-20 10:36:30

What causes brain damage and inflammation in severe cases of Gaucher disease? Little is known about the events that lead to brain pathology in some forms of the disease, and there is currently no treatment available – a bleak outlook for sufferers and their families. Now, scientists at the Weizmann Institute of Science have discovered a new cellular pathway implicated in Gaucher disease. Their findings, published today in Nature Medicine, may offer a new therapeutic target for the...

2013-12-18 11:47:56

Dopamine-producing cells die as result of cascade triggered by busy enzyme degradation cellular machines Scientists have identified the biological pathway that explains Gaucher disease (GD) patients' higher risk for developing Parkinson's disease (PD), according to research to be presented Tuesday, Dec. 17, at the American Society for Cell Biology annual meeting in New Orleans. GD is a hereditary disease in which the genes that encode the enzyme glucocerebrosidase are mutated, resulting...


Word of the Day
toccata
  • In music, a work for a keyboard-instrument, like the pianoforte or organ, originally intended to utilize and display varieties of touch: but the term has been extended so as to include many irregular works, similar to the prelude, the fantasia, and the improvisation.
This word is Italian in origin, coming from the feminine past participle of 'toccare,' to touch.
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