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Latest Rare diseases Stories

2014-06-17 12:26:05

Para-equestrian champion Rebecca Hart proves that having a disability doesn't derail your dreams. UNIONVILLE, Pa., June 17, 2014 /PRNewswire/ -- Born with a very rare genetic condition called hereditary spastic paraplegia (HSP) that keeps her from walking across a room unaided, six-time U.S. National Champion para-equestrian rider Rebecca Hart has set her sights on winning Gold at the 2014 AllTech FEI World Equestrian Games 2014 (WEG) in Normandy in August. After winning her sixth...

2014-06-11 12:28:34

Uniquely Positioned to Provide Big Data Analytics for Over 6,800 Rare Diseases BRANFORD, Conn., June 11, 2014 /PRNewswire/ -- BioXcel Corporation, announced today its first-in-class cloud based Pharma big-data analytics platform, PharmGPS(TM) now has a dedicated Orphan Disease suite to aid in the discovery, development, licensing and commercialization of drugs for the approximately 7,000 Rare and Ultra Rare Diseases. Partners will benefit from real-time multi-factorial analytics to...

2014-06-09 00:21:32

EPI-743 in phase 2B development for Leigh syndrome MOUNTAIN VIEW, Calif., June 9, 2014 /PRNewswire/ -- Edison Pharmaceuticals today announced that the FDA has granted Orphan Drug status to vatiquinone for the treatment of Leigh syndrome. Vatiquinone is the International Nonproprietary Name (INN) for Edison's EPI-743. The INN is a unique international name issued by the World Health Organization, which is used to identify the active pharmacological ingredient in a drug. This is...

2014-06-05 23:16:18

New officers focused on uniting the multiple system atrophy community to defeat the rare and terminal, neurodegenerative disorder. Expansion of the Board of Directors will be announced soon as important work continues. Charlotte, NC (PRWEB) June 05, 2014 The Multiple System Atrophy Coalition has elected new officers of its board of directors during their annual general meeting. Incorporated in 1999, The Multiple System Atrophy Coalition is a growing nonprofit 501 (c) 3 organization...

2014-06-04 08:33:48

WASHINGTON, June 4, 2014 /PRNewswire-USNewswire/ -- In a letter submitted today to the U.S. Food and Drug Administration (FDA), the National Organization for Rare Disorders (NORD) urged Commissioner Margaret Hamburg to give serious consideration to the concerns of the rare disease community when setting policy regarding official names for biologics, including biosimilars. With over 7,000 rare diseases identified and 30 million Americans affected, the patient population represented by NORD is...

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...

2014-06-02 08:31:12

Staff expanded in preparation for initiation of pulmonary and ophthalmic clinical trials DURHAM, N.C., June 2, 2014 /PRNewswire/ -- Parion Sciences, a company dedicated to the development of novel treatments for pulmonary and ocular diseases, today announced senior leadership and organizational changes. The changes include the following: -- Paul Boucher has been promoted to President and will lead the executive management team, business development and provide overall...

2014-05-29 23:09:19

Couple Builds upon Success of Last Year’s Event to Raise Awareness of Little-Known Disease at Lakeside Michigan Event Gull Lake, MI (PRWEB) May 29, 2014 After last year’s wildly successful event, Michelle Mackay, wife of retired Kellogg’s CEO David Mackay, and her family will host the second annual festive event to raise awareness and funds for the FSH Society on Saturday, July 5 at their lakeside home in Gull Lake, Michigan. Always an active member of her community, Michelle has...

2014-05-20 16:24:13

Focused Rowing Efforts & New Film Support Crucial ALS Research CHONSHOHOKEN, Pa., May 20, 2014 /PRNewswire/ -- For the next six months, Philadelphia will play host to another revolution, when the city's rowers come out in force to benefit Project A.L.S. Led by Carol Kleiner, a Philadelphia area native who was diagnosed with ALS, also known as Lou Gehrig's disease, the newly formed Carol's Crew will raise awareness and significant support for stem cell research, drug screening, and...

2014-05-16 08:26:28

- Data Demonstrate Positive Trends in Lung Function and Pulmonary Exacerbations - SOUTH PLAINFIELD, N.J., May 16, 2014 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that the results of a Phase 3 study of ataluren in patients with nonsense mutation cystic fibrosis (nmCF) were published in Lancet Respiratory Medicine. The results demonstrated positive trends in both the primary endpoint, lung function as measured by relative change in % predicted FEV(1) (forced...


Word of the Day
mallemaroking
  • Nautical, the visiting and carousing of sailors in the Greenland ships.
This word is apparently from a confusion of two similar Dutch words: 'mallemerok,' a foolish woman, and 'mallemok,' a name for some persons among the crew of a whaling vessel.