Quantcast

Latest Rare diseases Stories

2014-10-31 16:23:54

Board of Trustees Approves Integrated, Collaborative Approach WASHINGTON, Oct. 31, 2014 /PRNewswire-USNewswire/ -- The ALS Association announced on Thursday that the Board of Trustees has met and developed a long-term strategy for leveraging the Ice Bucket Challenge donations it has received to advance its integrated mission of leading the fight to treat and cure ALS through global research, care services and public policy. This will result in The Association tripling the amount of...

2014-10-30 16:29:19

LEXINGTON, Mass., Oct. 30, 2014 /PRNewswire/ -- Synageva BioPharma Corp. (NASDAQ: GEVA), a biopharmaceutical company developing therapeutic products for rare disorders, announced today third quarter 2014 financial results, and provided other business updates. Sebelipase alfa for LAL Deficiency The company recently started the rolling submission of a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for sebelipase alfa as a treatment for patients...

2014-10-29 23:16:31

The Centers for Disease Control and Prevention (CDC) selects DRT Strategies as the prime contractor to provide technical, database, and user experience support to the National ALS Registry. The web registry collects and provides information regarding ALS, sometimes called Lou Gehrig’s disease, and facilitates research into the causes of and potential cures for the disease. Arlington, Virginia (PRWEB) October 29, 2014 DRT Strategies, Inc. (DRT) (http://www.drtstrategies.com), a leading...

2014-10-27 08:28:39

MONTREAL, Oct. 27, 2014 /PRNewswire/ -- Clementia Pharmaceuticals, Inc. announced today the initiation of a Phase 2 extension study of palovarotene in patients with fibrodysplasia ossificans progressiva (FOP), a rare, severely disabling genetic disease characterized by painful, recurrent episodes of soft tissue swelling (flare-ups) and new abnormal bone formation. This process, known as heterotopic ossification (HO), occurs in muscles, tendons and ligaments, causing significant...

2014-10-27 08:26:15

Prospective blinded study demonstrates ability to accurately determine mutational status and monitor response to BRAF inhibitor therapy from urinary cell-free DNA SAN DIEGO, Oct. 27, 2014 /PRNewswire/ -- Trovagene, Inc., (NASDAQ: TROV) a developer of cell-free molecular diagnostics, today announced the publication of clinical study results in a peer-reviewed journal, Cancer Discovery, featuring the Company's precision cancer monitoring technology and its ability to non-invasively...

2014-10-23 20:29:41

TauRx Chairman Prof. Claude Wischik brings a clinical trial opportunity directly to caregivers and patients at breakfast event PHILADELPHIA and LONDON, Oct. 23, 2014 /PRNewswire/ -- MediciGlobal, a global leader in patient recruitment and retention for clinical trials, is supporting TauRx Therapeutics' Phase III clinical trial for behavioral variant Frontotemporal Dementia (bvFTD) by informing caregivers and patients about a "Meet the Experts" breakfast on Friday, October 24th, to...

2014-10-22 12:29:36

WASHINGTON, Oct. 22, 2014 /PRNewswire-USNewswire/ -- ALS ACT, The ALS Association, and the Translational Research Advancing Therapy ALS (TREAT ALS(TM)) Northeast ALS Consortium (NEALS) clinical trials network are pleased to announce a call for phase II clinical trial applications for novel, high-potential treatments in amyotrophic lateral sclerosis (ALS). The call for clinical study proposals is directed toward academic-industry partnerships, including pharmaceutical,...

2014-10-22 08:33:04

HAMILTON, N.J., Oct. 22, 2014 /PRNewswire/ -- Medical Diagnostic Laboratories, L.L.C., (MDL), a CLIA-certified, CAP-accredited laboratory which specializes in high complexity, state-of-the-art, automated DNA-based molecular analyses, announces that it is now offering new Cystic Fibrosis Testing which utilizes cutting edge technology, including next- generation DNA sequencing platforms. Cystic fibrosis (CF), or mucoviscidosis, is a genetically inherited multisystem disorder that...

2014-10-22 08:32:01

Architect of Novel Translational Rare Disease Programs at NIH Adds Depth to Existing Team of Rare Disease Experts SAN DIEGO and HONG KONG, Oct. 22, 2014 /PRNewswire/ -- aTyr Pharma ("aTyr"), an innovative rare disease therapeutics enterprise, announced today that rare disease expert John C. McKew, Ph.D., joined the company as vice president, research. Dr. McKew brings more than two decades of expertise in translational research, including key leadership positions at the National...

2014-10-21 16:28:41

First-Ever Product Submission for LAL Deficiency LEXINGTON, Mass., Oct. 21, 2014 /PRNewswire/ -- Synageva BioPharma Corp. (Synageva) (NASDAQ: GEVA), a biopharmaceutical company developing therapeutic products for rare disorders, announced today the start of a rolling submission of a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for sebelipase alfa as a treatment for patients with lysosomal acid lipase deficiency (LAL Deficiency), a rare genetic...


Word of the Day
maffling
  • To stammer.
  • Present participle of maffle, to stammer.
  • A simpleton.
The word 'maffle' may come from a Dutch word meaning 'to move the jaws' or a French word meaning 'having large cheeks'.