Spasmodic Dysphonia – A RARE voice condition
It is often described as an attack of laryngitis that “just didn’t go away” or an unexplained break in the voice, especially when certain words are produced. This is spasmodic dysphonia, a poorly understood condition in which the muscles that control the voice box involuntarily tighten. Spasmodic dysphonia appears to originate in the basal ganglia [...]
HAPPY THANKSGIVING – from all of us at RARE Project
Wishing you and your families the joy of the season ~ celebrating with your loved ones. We are grateful for your involvement in this important movement, coming together, to support one another. ~ Warmest Wishes from all of us at the RARE Project. Nicole, Dean, Jason, Amy, Christie, Jen, Lisa, Meme ~ Recommend on Facebook [...]
ULTRA Orphan Act – Fast Track Treatments for Ultra RARE Diseases
ULTRA Act UPDATE and REQUEST – Julia Jenkins, Everylife Foundation Congress is planning to introduce the Ultra-orphan Life-saving Treatments Act of 2012 or ULTRA Act to promote the discovery and development of safe and effective drugs and biologics to treat ultra-rare diseases. Your help is needed to ensure this legislation is successful! The ULTRA Act [...]
Clinical Development Program in LAL Deficiency
LAL Deficiency, a lysosomal storage disorder, has early and late onset phenotypes, also referred to as Wolman Disease or Cholesteryl Ester Storage Disease (CESD), respectively. There is no approved treatment for LAL Deficiency. Interventional research studies are currently recruiting patients for treatment with SBC-102. SBC-102 is Synageva BioPharma’s recombinant human lysosomal acid lipase being developed [...]
Be The Change at University of Minnesota
I want to operate in sheer honesty as CEXO (Chief Experience Officer) of the Be The Change campaign. For that reason, I want to disclose my love-hate relationship with the University of Minnesota (U of M) Hospital and Clinics. My individual relationship with the U of M has two sides, both humanly connected but medically [...]
Enter to win an iPod Touch through the EveryLife Art Contest!
Easy to enter! Just upload a photo of your art on RareArtist.org and you could win an iPod Touch! We accept art in all forms: Painting Photography Mixed Media Collages Pottery Sculptures PRIZES: Two Grand Prizes will be awarded in each age group: Children 5-11: $100 Visa Gift Card Teens 12-17: $250 Visa [...]
Cal-State Fullerton Students Champion RARE Disease
We are a group of five seniors at Cal-State University, Fullerton studying Communications – Public Relations. As part of our capstone Public Relations Management course, we were partnered with The R.A.R.E. Project (www.rareproject.org) to plan and implement a public relations campaign. Initially, we had little to no understanding of rare disease. We met with Nicole [...]
PATIENTSLIKEME AND R.A.R.E PROJECT UNITE TO FIND AND CONNECT ONE MILLION RARE DISEASE PATIENTS
Well received and well attended – Nonprofit and “Not Just for Profit” Announced Collaboration at the PartneringForCures Event! Excitement within the RARE Disease community around the PLM/RARE partnership. NEW YORK, NY – November 7, 2011 – Today, PatientsLikeMe and R.A.R.E Project, a nonprofit advocacy and support group for patients with all rare diseases, announce a [...]
ACTION ALERT: Wednesday RARE Disease Caucus Meeting needs your immediate attention!
Help ensure the first Rare Disease Caucus Breifing is a success! We want as many staffers to attend as possible to learn about the issues affecting the rare disease community. This is critically important and one of those moments where we need to come together and support what will ultimately benefit all those touched by [...]