FDA Orphan Drug Designation

There are currently about 7,000 orphans out there waiting to be adopted, to be supported, to be cured. Many of us don’t even know their names: Kawasaki. Porphyria. Pulmonary Arterial Disease.

Orphan Status is a designation given by the FDA to identify rare diseases with unmet medical needs but with potentially promising compounds currently being researched. Orphan drugs receive expedited FDA reviews and fast tracks to clinical trials, providing hope for cures, treatments or comfort.

>>>>>  Nearly 2,400 experimental compounds have orphan drug designations.

>>>>>  So far, only 367 of these orphan drugs have been approved by the FDA.

Experts agree, coordinated collaboration and resources on all fronts will be the key to finding a potential drug therapy for these orphan drugs. Collaboration is also essential for the rare diseases still searching for enough answers to even begin the orphan drug development stage.

You may ask, what can I do to help? What can my organization do?

Adopt An Orphan

• Pick an orphan disease with a compound in the works or a rare disease to adopt.

• Show your support and learn more about the disease, follow related organizations on Facebook or in the news.  One that we follow on Facebook is Two Hearts Rock and Global Genes Project.

• Donate your time, participate in a sponsored event, or fundraiser.

• “Wear That You Care” on World Rare Disease Day, February 29th. Wear “jeans” for “genes” and encourage your workplace, schools, and sport teams to do so too to raise awareness for the Global Genes Project.

At CorDynamics, we sponsored a fundraising event for Hope4Bridget and Batten Disease (a rare disease) as well as continue to work with our clients on promising compounds for the potential treatment of Pulmonary Arterial Hypertension (an orphan designated rare disease).

Working together, we will continue to adopt more of these orphan diseases and meet more unmet medical needs. To learn more about this issue and how you can help, jump into today’s blog hop sponsored by the R.A.R.E Project.

New Drug Therapy Would Give Hope4Bridget and Batten Disease

A rock concert for rare diseases? What’s to sing about?

Hope.

The Beat Batten & Rock Rare Disease concert given by Jesse Malin and the St. Marks Social in Chicago on July 23 will celebrate hope for awareness, new drug discoveries and specifically Hope4Bridget.

Bridget Kennicott was a spirited, rambunctious three-year old with delayed speech. Nothing rare about that. However her mother, an elementary school teacher and a breast cancer survivor, sensed there was something more. Then the seizures hit. After a marathon of doctors Bridget’s final diagnosis was devastating—Late Infantile Neuronal Ceroid Lipofuscinosis (LINCL) often referred to as Batten Disease. The children slowly deteriorate; the disease is fatal; there is no cure. Research is ongoing and some experimental drugs are currently in a limited clinical trial.

Through friends of friends, we heard about Bridget’s story and have followed her family’s journey to find treatments, causes, comfort and when all else fails—hope.

Developing lifesaving drug therapies for diseases of all magnitudes is what drives us day in and day out at CorDynamics. So why not carry that tune into our nightlife and back the band—so to speak?

Join us. If not that night, please join us in spirit, dedication and most of all—hope. Because where there is hope, there is always a glimmer of joy.