How A Bill Becomes a Law … It’s Complicated
We are often asked why we can’t quickly change the laws related to RARE disease therapy approval. The answer is that not only are politics always involved, it’s that the process is very complicated. This cartoon depicts what often happens in this process … what is needed seems very clear to us, but what is [...]
RARE Disease Drug Development … it’s complicated!
The development of any drug is a very complicated process … for RARE diseases the task is especially difficult due to the limited patient populations. Typically the disease is less well understood and there are fewer patients to access for testing to prove the drug works. We’re hoping this simplified overview of the process will [...]
Charities Unite Worldwide to Fund Research into Reversibility of Brain Damage Caused By Sanfilippo Syndrome
Charities Unite Worldwide to Fund Research into Reversibility of Brain Damage Caused By Sanfilippo Syndrome (RARE Project – Dana Point, CA) Rare Disease affects 350 million people worldwide, consisting of 7,000+ identified diseases and disorders. Currently less than 5% of all rare diseases have any type of therapy or treatment, and much of the early [...]
Wall Street Journal Reports – Do It Yourself Drug Development, With Some Help!
The effort by two families to buy and develop a drug that holds promise in treating Duchenne muscular dystrophy — described today in the WSJ — is the result of an innovative new model set up to support the burgeoning phenomenon of do-it-yourself drug development. Before the Seckler and Wicka families bought halofuginone, a drug that [...]
RARE DISEASE RESEARCH – Center for Applied Genomics
Rare Disease Research at the Center for Applied Genomics: Discovering the genetic variants that cause rare disease are essential to delivering better diagnoses, improved treatments, and preventive medicine. The most accurate and cost-effective method of achieving this goal is to sequence the genomes of affected patients, as well as their first-degree relatives. At the Center [...]
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 [...]
RE Children's Project launches innovative research consortium
Please follow this link to read about about our latest efforts to find a cure for rasmussen’s encephalitis. Thank you. Seth Wohlberg www.rechildrens.org Recommend on Facebook Share on Linkedin share via Reddit Share with Stumblers Tweet about it Subscribe to the comments on this post Print for later Bookmark in Browser Tell a friend
The Foundation for Prader-Willi Research (FPWR) Wins $50,000 RemedyMD Rare Disease Research Registry Donated by the Children’s Rare Disease Network (CRDN)
Announcement Comes After a Summer of Entrants Submitting Video Clips Telling Their Unique Stories in Exchange for Chance to Win Leading Life Sciences Research Software DANA POINT, CA/SALT LAKE CITY, UT, Oct. 17 /MarketWire/ –- In the spirit of Rare Disease Day, the Children’s Rare Disease Network (CRDN) received a donation from RemedyMD® (www.remedymd.com) of [...]
RemedyMD and the Children’s Rare Disease Network to Announce Winner of $50,000 Rare Disease Registry via Webinar, October 12th
Rare Disease Organizations Anxiously Await Results of Summer-Long Online Video Contest Giving Away Leading Life Science Research Platform Earlier this year we received a donation from RemedyMD (www.remedymd.com) of its RegistryOnDemand™ product specifically created for rare disease research organizations. We in turn announced a plan to give this research registry tool away to one lucky [...]
FINAL DAY TO ENTER TO WIN RARE DISEASE REGISTRY!
Today is the Final Day to Enter Videos to Win the RemedyMD Registry! This is it! You have until midnight tonight to submit your video just a few minutes in length, for your chance to win the R.A.R.E. Project and RemedyMD rare disease registry contest. The winner will receive a rare disease research software suite [...]