Gene therapy, still experimental but beginning to enter the clinic, attempts to utilize advanced molecular methods to treat and even reverse genetic diseases. The field started in earnest about 25 years ago and has had many setbacks along the way to its recent earliest successes. […]
7,000 Bracelets for Hope Continues throughout 2012 The 7,000 Bracelets of Hope campaign has already inspired hundreds of bracelet designers from all around the globe to join in helping champion the rare disease community by supporting this important cause! Bracelet donations are being accepted and […]
San Francisco Giants to host Rare Disease Fundraiser for MPS IV – with YouTube sensation Keenan Cahill!
The BIO International Convention has a tremendous tradition of connecting the Life Sciences Community of Stakeholders. Each year, BIO brings more opportunities to the table for patient advocacy groups to attend this event, garner exposure for their organization, and enjoy the benefits of the week […]
This year’s contest produced another amazing collection of beautiful artistic expression. We received many incredible and moving works of art in a variety of mediums, including paintings, drawings, quilts, photography, sculptures and glass work. Entries to this year’s contest grew by nearly 50% from our […]
A recent report released by PhRMA states that more drugs are currently in development for rare diseases than have been released in over 20 years since the Orphan Drug Act! The Pharmaceutical Research and Manufacturers of America (PhRMA) represents the country’s leading pharmaceutical research and […]