Pfizer Announces FDA Approval of ELELYSO™ for Type 1 Gaucher Disease and Personal Support Program For Patients
On May 1, 2012, Pfizer announced the FDA approval of ELELYSO™ (taliglucerase alfa), an enzyme replacement therapy (ERT) for adults diagnosed with a rare genetic disorder called type 1 Gaucher disease. ELELYSO™, the first FDA-approved plant cell-based ERT for Gaucher disease, is also recognized for being acquired through a genetically engineered manufacturing system. This news [...]
Remi Savioz Glut1 Foundation Virtual Run: May 6
Sunday, May 6, 2012 Running for the Remi Savioz Glut1 Foundation! Every mile “Makes a Difference” Every Step is an opportunity to finding a cure. On Sunday, May 6, 2012 James Dylan’s mom June, along with many other families, will be participating in an important challenge for her son and the Remi Savioz Glut1 [...]
‘Rock the Cure’: Rocking Out for Dravet Syndrome and Eosinophilic Esophagitis
Bands will ‘Rock the Cure’ this weekend by Kelli Easterling Richmond County Daily Journal Rockingham, NC – Reported on April 10, 2012 Local contemporary Christian bands have joined forces to “Rock the Cure” this Saturday, while “rocking out” on two stages in the Winn Dixie parking lot in Rockingham from 11 a.m until 4 p.m. “There [...]
Rickshaw Run 2012: Cool Runnings for Sanfilippo Syndrome
On March 22, R.A.R.E. Project announced the amazing endeavors of Joe and Paul, two adventurists who planned to participate in the RickShaw Run to support Sanfilippo Syndrome with R.A.R.E. Project as their charity of choice. On April 6 - Joe and Paul, a.k.a. team Cool Runnings, began their adventures in India to fight for the [...]
A Fifth Season…Brightening the Landscape of Batten Disease
Rare Disease Event: Three Foundations Form First Chicago Partnership To Fight LINCL-Batten Disease Jasper Against Batten, Hope 4 Bridget and Noah’s Hope To Collaborate with Partnership For Cures One man ran across the United States to help children with Batten disease. Will you drive across town to help? Please join us on May [...]
Psychotherapist Joins R.A.R.E. Project Team as Guest Blogger
R.A.R.E. Team Guest Blogger: Vanessa Lemminger, M.A., IMF We would like to welcome you to our new R.A.R.E. Project Team Guest Blogger, Vanessa Lemminger! Vanessa Lemminger is a psychotherapist and bereavement specialist working in private practice in San Diego. As a State of California Board of Behavioral Science Registered Marriage and Family Therapy Intern IMF #68894, [...]
Mitochondrial Disease: Jeans for Genes
An Amazing Response To a Simple Campaign Raising Awareness for Rare Disease Day and Mitochondrial Disease Many of us struggle with not knowing how we can make an impact on Rare Disease. Whether we are faced with the day-to-day difficulties of a rare disease itself, or we are passerby’s watching others’ demanding fight – [...]
Behind the Scenes at the Genetic Disease Foundation
Genetic Disease Foundation welcomes Scientific Advisory Board Members We understand that genetics plays a big role in the world or rare disease. We know that genetic testing assists physicians in determining concrete diagnoses. And in order to effectively treat an individual for their diagnosis, we must understand the genetic makeup of that particular disease [...]
MarbleRoad: A Storybook Success
Helping To Raise Awareness MarbleRoad, a non-profit based in DC wanted to make a creative impact to raising awareness to families in need. Their mission: ….” to connect people who have complex illnesses with the resources they need to help them improve their lives.” And with the help of RAREProject and others, MarbleRoad accomplished their [...]
Change is Needed at the FDA
“Breakthrough technologies deserve a breakthrough in the way the FDA evaluates them.” - Andrew Von Eschenbach Mr. Von Eschenbach couldn’t have stated it any clearer. On February 14, the Wall Street Journal posted an on-line article by Mr. Eschenbach titled: Medical Innovation: How the U.S. Can Retain Its Lead, The FDA should approve drugs based on [...]