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NORD to Honor Rare Disease Innovators
Date:5/11/2010

ultiple sclerosis.  This is the first drug approved by the Food and Drug Administration (FDA) for this purpose.

Allos Therapeutics for development of FOLOTYN® (pralatrexate injection) for use as a single agent for treatment of patients with relapsed or refractory peripheral T-cell lymphoma (PTCL).  This is the only drug approved by FDA for this condition.

Lundbeck Inc. for development of Sabril® (vigabatrin) Oral Solution for treatment of infantile spasms in children ages one month to two years.  This is the first drug approved in the U.S. to treat this disorder, which is characterized by a severe type of seizure.

Novartis for development of Ilaris® (canakinumab) for treatment of patients aged four and older with Muckle-Wells syndrome or familial cold auto-inflammatory syndrome (FCAS), both of which are forms of "CAPS," a group of rare, debilitating auto-inflammatory disorders.

Shire for development of VPRIV™ (velaglucerase alfa for injection) to treat children and adults with Type 1 Gaucher disease, a rare and disabling genetic disorder that occurs as a result of an enzyme deficiency.  VPRIV™ provides a treatment alternative for patients at a time when another therapy for this disease is in short supply.  

About NORD:

The National Organization for Rare Disorders (NORD) was established in 1983.  It is a nonprofit organization representing all Americans affected by rare diseases.  NORD's services include advocacy on public policy issues; education and information for patients, families, medical professionals, and the public; patient assistance programs to help patients obtain certain medications they could not otherwise afford; mentoring for other patient organizations; and r
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SOURCE National Organization for Rare Disorders (NORD)
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