GOOD MORNING EVERYONE.
THROUGH OUT OUR BEHIND THE MYSTERY SERIES, WE'VE LEARNED
ABOUT VARIOUS RARE AND GENETIC DISEASES LIKE LAL DEFICIENCY AND
PULMONARY FIBROSIS, AS WELL AS INITIATIVES LIKE THE GLOBAL
GENES PROJECT.
TODAY, WERE DISCUSSING ORPHAN DRUGS.
AN ORPHAN DRUG IS A MEDICATION THAT HAS BEEN DEVELOPED
SPECIFICALLY TO TREAT A RARE MEDICAL CONDITION.
THE CONDITION ITSELF BEING REFERRED TO AS AN ORPHAN
DISEASE, HENCE, ORPHAN DRUG.
RECENTLY WE ATTENDED A VERY SPECIAL CONFERENCE TO LEARN MORE
ABOUT THIS UNIQUE AREA OF MEDICINE.
TAKE A LOOK.
(MUSIC).
HELD IN WASHINGTON D.C.
AT THE MANDARIN ORIENTAL, THE 2013 WORLD ORPHAN DRUG CONGRESS
IS THE WORLDS LARGEST ORPHAN DRUG CONFERENCE AND EXPO.
(MUSIC).
THE GOAL OF THE WORLD ORPHAN DRUG CONGRESS USA IS REALLY TO
BE THE FACE OF THE ORPHAN DRUG INDUSTRY, THIS IS WHERE EVERY
YEAR, DIFFERENT HOLDERS PATIENTS GROUPS PHARMA INDUSTRY AND
INVESTORS,PAYERS ETC.
THIS IS WHERE THEY CAN COME YEAR AND
YEAR TO MEET EVERYONE AND REALLY JUST TO BE ABLE TO BRAINSTORM
REALLY TO BE ABLE TO JUST FIGURE OUT WHAT ARE THE CHALLENGES AND
WHAT ARE THE STRATEGIES TO OVERCOME THESE CHALLENGES SO
THAT WE CAN EXPEDITE ORPAHN DRUGS TO THE RARE DISEASE
PATIENTS WHO NEED THE ORPHAN DRUGS.
(MUSIC).
THERE ARE A FEW PATIENTS, LESS THAN 10,000 IN THE WORLD WITH
ORPHAN DISEASES, THAT THERE SHOULD BE A PRODUCT AVAILABLE
FOR IT AND SO I HOPE THAT MANY MORE COMPANIES WILL ACTUALLY
DEVELOP THOSE PRODUCTS FOR THE FIVE THOUSAND, SIX THOUSAND
DIFFERENT GENETIC DISEASES THAT ARE THERE AND ONLY THREE HUNDRED
ARE ABLE TO BE TREATED.
(MUSIC).
FOR THOSE PARTICIPATING IN THE EVENT, ITS A CHANCE TO INTERACT
WITH MANY DIFFERENT ASPECTS OF THE ORPHAN DRUG COMMUNITY.
WE FIND IT A GREAT WAY TO COLLABORATE AND PARTNER WITH NOT
ONLY OUR INDUSTRY COLLEGES BUT ALSO PUBLIC POLICY, ADVOCACY AND
OTHER INTERESTED STAKE HOLDERS.
(MUSIC).
AND FOR THOSE ATTENDING THE WORLD ORPHAN DRUG CONGRESS, THE
REASONS FOR BEING HERE ARE EQUALLY COMPELLING.
THIS EVENT IS REALLY IMPORTANT BECAUSE IT BRINGS TOGETHER ALL
DIFFERENT SECTORS OF THE RARE DISEASE COMMUNITY AND ALLOWS
PEOPLE TO SHARE THEIR KNOWLEDGE, THEIR EXPERTISE, CHALLENGES
AND SOLUTIONS.
SO I THINK ITS A REALLY WONDERFUL WAY TO COME TOGETHER
AND HAVE ALL THAT EXPERTISE IN ONE PLACE.
(MUSIC).
COMPANIES LIKE OURS ARE INVOLVED WITH EVENTS SUCH AS THESE
BECAUSE THEY BRING TOGETHER A WIDE VARIETY OF DIFFERENT
COMPANIES OF DIFFERENT BACKGROUNDS FOCUSED ON NEW
THERAPEUTICS IN RARE DISEASES.
WE ALL SHARE A COMMON GOAL HERE IN TRYING TO BRING TO PEOPLES
ATTENTION THE FACT THAT THERE ARE DISORDERS AND DISEASES THAT
NEED ATTENTION AND THAT INCREASINGLY AS SCIENCE
PROGRESSES WE'RE GOING TO BE ABLE TO TARGET MORE AND MORE,
SPECIFIC CURES FOR SPECIFIC DISEASES AND THE CONFERENCE HAS
BEEN REALLY EXCELLENT IN STARTING TO FOCUS PEOPLES
ATTENTION ON THAT.
WHETHER THERES SIMPLY ATTENDING OR HERE PRESENTING, THOSE
INVOLVED UNDERSTAND THIS COMMERCIALLY FOCUSED EVENT FOR
THE ADVANCEMENT OF RARE DISEASE RESEARCH AND ORPHAN DRUG
DEVELOPMENT IS AN IMPORTANT EVENT.
WE ALL WORK IN THE RARE DISEASE SPACE BUT WE WORK IN OUR OWN
LITTLE CORNERS OF THE WORLD, THIS ACTUALLY BRINGS US ALL
TOGETHER WHERE WE CAN MEET PEOPLE FACE TO FACE.
WHAT I REALLY HOPE IS THAT WHEN PEOPLE COME TO THIS EVENT, WHAT
THEY ARE GOING TO TAKE AWAY IS THAT WE'RE ALL IN THIS TOGETHER.
THAT EVERYBODY HAS A ROLE TO PLAY AND HOW WE CAN ALL WORK
TOGETHER TO HAVE BETTER PATIENT OUTCOMES.
(MUSIC).
SO GREAT TO SEE HOW MANY PEOPLE AND ORGANIZATIONS ARE WORKING SO
HARD TO FIND ANSWERS TO THE MYSTERIES BEHINDS THESE RARE AND
GENETIC DISEASES, NOW BE SURE TO WATCH FOR MORE IN OUR SERIES
"BEHIND THE MYSTERY RARE AND GENETIC DISEASES" AND OF COURSE
CHECK OUT RELATED INFORMATION ON OUR WEBSITE AT THE
BALANCINGACT.COM/RARE.