Latest Rare diseases Stories

What Is The Cost Of Rare Diseases Such As Friedreich's Ataxia?

2013-02-28 14:16:28

What is the cost of rare diseases such as Friedreich's Ataxia? By analyzing direct and indirect costs of care research in BioMed Central's open access journal Orphanet Journal of Rare Diseases calculated that conservatively this rare disease costs between £11,000 and £19,000 per person per year. Proper understanding resource allocation is important in minimizing the effect of Friedreich's Ataxia on people's lives while maximizing their quality of life. Diseases are classified as 'rare'...

Assay Depot And Rare Genomics Institute Award Prizes To Study 26 Rare Diseases

2013-02-28 12:27:30

More Than $500,000 Worth of Services, Reagents, and Prize Money SAN DIEGO and ST. LOUIS, Feb. 28, 2013 /PRNewswire/ -- In recognition of Rare Disease Day, Assay Depot and Rare Genomics Institute (RGI) today announced the winners of the first ever Rare Disease Science Challenge: BeHEARD (Helping Empower and Accelerate Research Discoveries). In total, more than $500,000 worth of research services will be awarded to study 26 different rare diseases, ranging from Alkaptonuria to...

Quantum Cures Announces Breakthrough Approach for Researchers Seeking Cures for Orphan and Rare Diseases

2013-02-28 08:30:43

Collaborative donations of computing cycles to enable more in-depth research; goal is speeding discovery of small molecules for use in new medicines and vaccines EXTON, Pa., Feb. 28, 2013 /PRNewswire/ -- A new effort, being introduced today, aims to make it possible for medical researchers to develop small molecules that can be used to prevent, treat and find cures for a wide range of debilitating diseases by enlisting the help of volunteers around the world and using their idle...

Shire Supports Rare Disease Day 2013

2013-02-28 04:21:45

LEXINGTON, Massachusetts, February 28, 2013 /PRNewswire/ -- - Global Initiatives Underscore Company's Long-Term Commitment to the Rare Disease Community Shire plc (LSE: SHP, NASDAQ: SHPG) today announced its support of Rare Disease Day, joining patients, healthcare providers, and patient organizations around the world to support a day that focuses attention on rare diseases as a public health issue, and highlights the need for more understanding and awareness about...

The ALS Association Kicks Off 2013 Walk to Defeat ALS® Season

2013-02-26 16:26:34

170 local events scheduled in communities nationwide to fight Lou Gehrig's Disease WASHINGTON, Feb. 26, 2013 /PRNewswire-USNewswire/ -- The ALS Association 2013 Walk to Defeat ALS® season begins this week and runs through November with 170 local events in communities across the nation. These events provide a wonderful opportunity to raise awareness about Lou Gehrig's Disease and to support the mission of The ALS Association, the only national non-profit organization fighting...

The ALS Association Urges U.S. Food and Drug Administration to Expedite Drug Development and Approval Process at First-Ever ALS-Specific Public Hearing

2013-02-25 16:26:17

WASHINGTON, Feb. 25, 2013 /PRNewswire-USNewswire/ -- Today, leaders from The ALS Association, in addition to patients, caregivers, physicians, scientists and other concerned individuals, addressed a panel of representatives from the U.S. Food and Drug Administration (FDA) as the agency conducted its first-ever public hearing specific to amyotrophic lateral sclerosis (ALS). The Association urged the FDA to partner with the ALS community to help expedite the drug development and...

Neuralstem Grants Licenses For Central Nervous System Therapy Surgical Devices To Cedars-Sinai Medical Center

2013-02-20 08:29:53

ROCKVILLE, Md., Feb. 20, 2013 /PRNewswire/ -- Neuralstem, Inc. (NYSE MKT: CUR) announced that it has granted licenses to intellectual property surrounding its spinal cord delivery platform, floating cannula, and method for delivering therapeutic agents to the spinal cord to Cedars-Sinai Medical Center, a non-profit academic medical center located in Los Angeles, CA. The license agreements grant Cedars-Sinai Medical Center the non-exclusive right to use the licensed intellectual...

Shire's VPRIV® Wins First Ever "Novel Treatment Award" From Lysosomal Disease Network at Their 9th WORLD Symposium

2013-02-19 08:32:40

LEXINGTON, Massachusetts, February 19, 2013 /PRNewswire/ -- Shire plc (LSE: SHP, NASDAQ: SHPG), announces that the Lysosomal Disease Network (LDN) presented Shire Human Genetic Therapies (HGT) with the first ever Novel Treatment Award for VPRIV on February 14th, at its 9th Annual WORLD Symposium. The LDN have launched this new award, which will be presented annually, to recognize new drugs or therapies that have made a considerable contribution in the area of lysosomal...

Scioderm's Investigational New Drug (IND) Application for SD-101 for Treatment of Epidermolysis Bullosa (EB) Allowed by the FDA

2013-02-13 20:21:47

RALEIGH, N.C., Feb. 13, 2013 /PRNewswire-iReach/ -- Scioderm announced that the US Food and Drug Administration (FDA) has reviewed and allowed the investigational new drug (IND) application for SD-101 to proceed. SD-101 is a topical treatment being developed for the treatment of Epidermolysis Bullosa (EB). The Company plans to initiate a Phase 1 study in the coming months, with initiation of the Phase 2B/3 study in EB patients to occur in the second half of 2013. EB is a rare...

Novel Protein May Help Detect Lou Gehrig's Disease And Dementia

2013-02-12 20:29:59

Researchers at Mayo Clinic have discovered an abnormal protein that accumulates in the brains of many patients affected with two common neurodegenerative disorders — amyotrophic lateral sclerosis, also known as ALS or Lou Gehrig's disease, and frontotemporal dementia. They say their findings have uncovered a potentially new therapeutic target and biomarker that would allow clinicians to confirm diagnosis of the diseases. The study is published online today in the journal Neuron. The Mayo...