Vertex’s 2-Drug Cystic Fibrosis Pill Shows Promise


Vertex Pharmaceuticals said on Tuesday that a combination of two of its drugs had successfully treated cystic fibrosis in closely watched clinical trials, potentially clearing the way for approval of a new option for nearly half the patients with the genetic disease. The drug combination, […]

Canada Joins International Web Portal for Rare Disease Research


Canada is the first country in the Americas to launch an international Web portal designed to improve research and treatments for rare diseases, Health Data Management reports. Portal Details The international portal– called Orphanet– is led by a consortium overseen by the French National Institute of Health and […]

More Patients Help Drug Companies Pass Valley of Death


There’s no question the pharmaceutical industry has boatloads of cash. Global sales of prescription drugs total nearly $1 trillion annually, according to IMS Health, a Connecticut-based market research firm. Drug companies spend about $200 billion per year on researching and developing new drugs, according to […]

Rhode Island Adds Immune Deficiency to Newborn Screening Test


Rhode Island has expanded its program of infant screening to include a rare immune deficiency. The state Health Department said all newborns will be screened for severe combined immunodeficiency, or SCID, beginning Friday. The disease causes dangerously low levels of the white blood cells that […]

Texas Man Is Fourth In U.S. To Die From Variant Creutzfeldt-Jakob Disease


AUSTIN, Texas, June 5 (Reuters) – A Texas man has died of the fatal brain illness variant Creutzfeldt-Jakob Disease, the fourth person to die of the disease in the United States, according to the Centers for Disease Control. CJD is a rare, degenerative, fatal brain […]

NBC Reports: Kids With Rare Disease May Hold Secret to Stopping Viruses


A brother and sister with an extremely rare genetic condition have a stunning ability to fight off viruses— an ability that could lead to a new family of antiviral drugs. Their bodies stop viruses from building a protective shell, which leaves viruses “naked” and vulnerable, […]

GLOBAL GENES™ Launches “RARE Toolkits” as Premier Educational Resource for the Rare Disease Community

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Series of RARE Disease Toolkits Enable Advocates, Patients and Caregivers to Empower Themselves and Inspire Others With 1 in 10 Americans living with rare diseases, the need for access to powerful, actionable information has never been greater. Global Genes™ is proud to launch RARE Toolkits, […]