Rare Disease in the News

Rare diseases and individualised approaches
PMLiVE (blog)
Patients with rare diseases can often end up on treatment pathways for more common conditions, which are not often suited to their unique needs. Within primary care, physicians may only encounter one patient with a specific rare disease in their entire ...

Shire inks a $225M rare disease deal on the eve of life with AbbVie
For Shire, the move dovetails with both its broad base of work in rare diseases and its efforts in Hunter's. The company's Elaprase, FDA approved in 2006, was the first marketed treatment for the disorder, and Shire is in the midst of Phase II with SHP ...

Shire expands rare disease reach
Medical Marketing and Media
Shire, which is soon to be AbbVie, has finalized a deal with US biotech ArmaGen. The $225-million deal gives Shire worldwide commercialization rights to the experimental enzyme replacement therapy AGT-182, which could be used to treat what the firms ...
Shire, ArmaGen ink worldwide licensing pact for enzyme replacement therapypharmabiz.com
ViroPharma Incorporated: Shire Enters Strategic Licensing and Collaboration ...The Wall Street Transcript
Shire In $225 Mln Licensing, Collaboration Deal With ArmaGen For AGT-182NASDAQ

all 27 news articles »

7 Year Old With Rare Disease Throws Out First Pitch At Hillcats Game
Lynchburg, VA - A 7 year old boy from Gretna threw out the first pitch at Wednesday night's Lynchburg Hillcats game. He suffers from a rare disease. Perthes affects eight in every 100,000 children in the U.S. and Andrew Logwood is one of them. The ...

Drug Discovery & Development

PSI, AOP Orphan Collaborate on Global Rare Disease Trial
Drug Discovery & Development
Polycythemia vera (PV) is a Rare Disease of the blood-building cells in the bone marrow primarily resulting in a chronic increase of red blood cells (erythrocytes), thereby having an adverse impact on blood rheology. Circulatory disorders such as ...

Network of experts join forces to fight rare disease
Collaboration between research groups is key in tackling rare diseases such as auto-immune disease Myasthenia Gravis (MG). Indeed, the rarity of the disease means that it can be difficult to collect enough samples of blood and tissues to perform ...

and more »

Home News New therapy helps London teen with 'ultra-rare'...
London Community News
For more than two years Gacic and his mom Verica had travelled from London to Toronto every week to take part in a clinical trial for a new treatment for an “ultra-rare” genetic disease — only about 50-100 Canadians are afflicted — with no cure. So ...

Sage takes to the FDA fast track with its lead rare disease drug
Fresh off grossing $103.5 million in an overstuffed IPO, Cambridge, MA's Sage Therapeutics ($SAGE) has secured the promise of a speedy FDA review for its top prospect, a treatment for a rare seizure disorder. The agency has granted its fast-track ...

and more »


Medical Mysteries: New Network Will Help Rare Disease Detectives
They baffle even the finest specialists: rare diseases that affect only a handful of people and that are often caused by extremely rare genetic mutations. Now the National Institutes of Health is setting up a network of research centers where doctors ...
NIH chooses Vanderbilt for rare diseases networkThe Tennessean
US agency taps medical centers to investigate rare diseasesReuters
NIH Picks Vanderbilt, 5 Other Universities To Be Part Of Rare Diseases NetworkBioResearch Online (press release)
National Institutes of Health (press release)
all 113 news articles »

PMI taking donations for baby with rare disease
Jakarta Post
The Surakarta chapter of the Indonesian Red Cross (PMI), in cooperation with Surakarta's city-owned Moewardi hospital, has taken the initiative to support one-year-old Calista Ramadani, who was diagnosed with Alagille syndrome. PMI will open a bank ...