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Latest Rare diseases Stories

2014-08-07 23:12:05

Tom Looney, diagnosed with MSA in 2008, worked closely with tech industry icons, including Steve Jobs. His unique experiences and talents will now be utilized to represent patients and increase awareness for this rare and terminal neurological disorder. Wilmington, NC (PRWEB) August 07, 2014 The Multiple System Atrophy (MSA) Coalition, an organization dedicated to serving patients, caregivers and families touched by MSA, has announced the appointment of veteran tech industry exec Tom...

2014-08-06 16:28:42

Input will help shape the role of public contributions to orphan drug approvals OTTAWA, Aug. 6, 2014 /CNW/ - The Honourable Rona Ambrose, Minister of Health, announced today that the Government of Canada has launched a pilot project targeting patient input from Canadians with rare diseases to help inform future reviews of orphan drugs. As announced in October of 2012, Health Canada has been developing an Orphan Drug Framework to spur innovation and research into new treatments...

2014-08-05 12:31:36

A nine-year study recently released proves the need TOWSON, Md., Aug. 5, 2014 /PRNewswire/ -- The Immune Deficiency Foundation (IDF) commends the states of Maine and Rhode Island for adding Severe Combined Immune Deficiency (SCID) to their states' newborn screening panels, effective August 1, 2014. These states join 21 other states currently screening newborns for SCID, ensuring over two thirds of all babies in the U.S. are now being screened. SCID is a type of primary...

2014-08-05 08:33:19

SAN FRANCISCO, Aug. 5, 2014 /PRNewswire/ -- Perlstein Lab PBC, a San Francisco-based public benefit corporation whose mission is precision orphan drug discovery, just closed a $2 million seed round. The round was co-led by the clinical-stage orphan drug developer Retrophin, Inc. and the Wilsey family, which is personally affected by an orphan disease. http://photos.prnewswire.com/prnvar/20140805/133384 One in 12 people suffer from an orphan disease, yet 95% of orphan diseases have...

2014-08-01 23:07:34

In her new role, Sharon will focus on advocating for and supporting MSA families as well as on educating healthcare professionals unfamiliar with this rare neurological disorder. La Coste, TX (PRWEB) August 01, 2014 The Multiple System Atrophy Coalition, a growing nonprofit 501 (c) 3 organization dedicated to serving patients, caregivers and families affected by the neurological disorder Multiple System Atrophy (MSA), announced the appointment of Sharon Kramer Sutton of La Coste, TX to...

2014-07-31 23:17:56

In his new role, Dr. Kellerman will be representing the needs of current caregivers as well as focusing on educating healthcare professionals unfamiliar with this rare neurological disorder. New London, IA (PRWEB) July 31, 2014 The Multiple System Atrophy Coalition, a growing nonprofit 501(c) 3 organization dedicated to serving patients, caregivers and families affected by the neurological disorder Multiple System Atrophy (MSA), announced the appointment of Dr. Larry Kellerman of New...

2014-07-31 08:34:32

Acceptance of Regulatory Submission Triggers $10 Million Milestone Payment to Auxilium CHESTERBROOK, Pa., July 31, 2014 /PRNewswire/ -- Auxilium Pharmaceuticals, Inc. (NASDAQ: AUXL), a specialty biopharmaceutical company, today announced that the Company will receive a $10 million regulatory milestone payment from its partner Asahi Kasei Pharma Corporation (Asahi Kasei). The payment is due because of the successful submission of a regulatory application to the Japanese Pharmaceutical...

2014-07-30 16:29:33

LEXINGTON, Mass., July 30, 2014 /PRNewswire/ -- Synageva BioPharma Corp. ("Synageva") (NASDAQ: GEVA), a biopharmaceutical company developing therapeutic products for rare disorders, today reported second quarter 2014 financial results, and provided other business updates. Second Quarter 2014 Financial Results For the quarter ended June 30, 2014, Synageva reported a net loss of $50.2 million compared to a net loss of $22.1 million for the corresponding quarter of the prior year....

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 08:30:28

ArmaGen to Receive up to Approximately $225 Million Including Initial Upfront Payment of $15 Million in Cash and Equity, an Additional Equity Investment, R&D Funding, Development and Sales Milestones, in Addition to Future Royalties CALABASAS, Calif., July 23, 2014 /PRNewswire/ -- ArmaGen, a privately held biotechnology company focused on developing revolutionary therapies to treat severe neurological disorders, announced today that it has entered into a worldwide licensing and...


Word of the Day
bodacious
  • Remarkable; prodigious.
  • Audacious; gutsy.
  • Completely; extremely.
  • Audaciously; boldly.
  • Impressively great in size; enormous; extraordinary.
This word is probably from the dialectal 'boldacious,' a blend of 'bold' and 'audacious.'
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