THIS WEEK MARKS RARE DISEASE DAY, SHINING A LIGHT ON THE BATTLE ONE IN TEN PEOPLE ENDURING AGAINST ILLNESSES OFTEN INCURABLE AND UNDERSTUDIED.

ONE OF THOSE RARE ILLNESSES IS EPIDERMOLYSIS BULLOSA, OR E.B.

A GENETIC SKIN DISEASE THAT CAUSES BLISTERING, LEAVING PATIENTS TO LIVE CONSTANTLY WRAPPED UP IN BANDAGES.

THE LIVES OF E.B.

PATIENTS ARE THE FOCUS OF A NEW DOCUMENTARY NOW STREAMING ON NETFLIX ENTITLED, MATTER OF TIME.

HARI SREENIVASAN SPEAKS TO E.B.

EXPERTS DR.

JEAN TANG AND MICHAEL HUND ABOUT THE STRUGGLE AND THE RESILIENCE OF THOSE GRAPPLING WITH THIS CONDITION.

>> THANKS SO MUCH.

DR.

JEAN TANG AND MICHAEL HUND, THANK YOU BOTH FOR JOINING US.

DR.

TANG, I WANT TO START WITH YOU YOU'RE A PROFESSOR OF DERMATOLOGY AT STANFORD UNIVERSITY AND YOU ARE FOCUSED ON RARE GENETIC ZIN DISEASES INCLUDING EPIDERMOLYSIS BULLOSA, THE SKIN DISEASE FEATURED IN THIS DOCUMENTARY.

AND FIRST OF ALL, I GUESS WHAT IS IT?

WHAT DOES IT DO TO PEOPLE?

>> EPIDERMOLYSIS BULLOSA, OR E.B., IS A GENETIC SKIN DISEASE.

IS RARE THANKFULLY.

THESE UNFORTUNATE PATIENTS INHERIT A GENETIC MUTATION, A SINGLE GENETIC LETTER CHANGE THAT MAKES THE DIFFERENCE BETWEEN ME AND YOU AND THEM WHERE FRAGILE SKIN THAT'S OFTEN WOUNDED, PAINFUL.

MANY CAN'T GO TO SCHOOL, CAN'T RUN, CAN'T PLAY SOCCER, AND CAN'T HAVE A NORMAL QUALITY OF LIFE THAT A CHILD DESERVES BECAUSE OF THIS SINGLE ONE GENETIC MUTATION.

>> WHAT'S HAPPENING TO THEIR SKIN?

>> RIGHT.

SO THE GENETIC MUTATION CHANGES A PROTEIN, SO THE PROTEIN DOESN'T -- ISN'T ABLE TO STAPLE THE TOP LAYER OF THE SKIN TO THE BOTTOM LAYER OF YOUR SKIN.

WITH ANY GENTK FRICTION OR TRAUMA, THE SKIN SHEARS APART AND THERE ARE BLISTERS AND WOUNDS.

THESE KIDS ARE COVERED IN WOUND DRESSINGS FROM HEAD TO TOE TO TRY TO PROTECT THEIR SKIN.

>> MICHAEL, THE FILM IS CALLED MATTER OF TIME, AND I WATCHED IT, YOU KNOW, PARTLY BECAUSE I HAVE A CHILD OF THE '90s FROM SEATTLE, OH MY GOSH, EDDIE VEDDER FROM PEARL JAM IS INVOLVED IN SOMETHING.

LET ME FIND OUT MOORE, RIGHT.

I AM WATCHING THIS WHAT COULD BE A CONCERT VIDEO, BUT IN BETWEEN YOU SEE HOW COMMITTED HE AND HIS WIFE JILL HAVE BEEN TO TRYING TO FIGURE OUT A CURE FOR E.B.

WHAT MOTIVATED THEM IN THE FIRST PLACE?

>> YEAH, SO JILL AND ED ARE HERCULEAN, RIGHT, WHAT THEY HAVE GIVEN IN THEIR LEADERSHIP, THEIR PLATFORM SHINING A LOT ON THIS COMMUNITY AND REALLY THE INSPIRATION WAS JILL HAD A CHILDHOOD FRIEND WHO HAD A SON WITH EPIDERMOLYSIS BULLOSA, AND, YOU KNOW, WHAT WE HOPE THE FILM CONVEYS IS WHEN YOU MEET SOMEONE WITH E.B.

YOU QUICKLY LEARN A LOT ABOUT BRAVERY AND COURAGE AND RESILIENCE.

AND CERTAINLY IT HAD THAT IMPACT ON JILL AND ED.

THEY SAID WE HAVE GOT A PLATFORM, WE HAVE GOT A VOICE, WE'VE GOT A STAGE.

LET'S SHED A LIGHT ON THIS COMMUNITY.

WE STARTED WITH FUNDING A HANDFUL OF RESEARCHERS.

THAT'S EXPANDED TO 200 PROJECTS IN 22 COUNTRIES, $80 MILLION RAISED ACROSS THE PLANET, TWO CLINICAL TRIALS TO MORE THAN 50 CLINICAL TRIALS.

IN THE LAST TWO YEARS ALONE THANKS TO BRILLIANT FOLKS LIKE DR.

JEAN TANG, WE HAVE SEEN THREE FDA APPROVALS FOR EPIDERMOLYSIS BULLOSA, WHICH GIVES US HOPE BECAUSE IF YOU THINK ABOUT E.B., IT'S ONE OF 10,000 OR MORE RARE DISEASES, 400 MILLION PEOPLE ON THE PLANET, YET 95% DON'T HAVE ANY FDA APPROVED TREATMENTS.

WE HAVE DONE THAT THREE TIMES IN THE LAST TWO YEARS.

THOSE TREATMENTS WE WANT TO -- SO THAT'S THE GOAL.

>> DR.

TANG, THERE ARE SEVERAL STORIES OF DIFFERENT PEOPLE, DIFFERENT PATIENTS OF DIFFERENT AGES AND WHAT THEY ARE GOING THROUGH.

ONE IS OF ROWAN HALLER.

>> WHEN SHE STARTED HAVING WOUNDS APPEARING, IT QUICKLY BECAME BASICALLY LIKE A NIGHTMARE.

AND THE WORST DAY OF MY LIFE.

THAT'S WHEN A DOCTOR CAME IN AND SAID SHE EITHER HAS A SERIOUS INFECTION OR SHE HAS SOMETHING CALLED EPIDERMOLYSIS BULLOSA.

AND HE SAID IT SO QUIETLY AND HE SAID, AND IT WILL BE FOR LIFE.

>> TELL US HOW DO CHILDREN LIKE ROWAN GET THROUGH THE DAY?

WHAT ARE THE ACTS OF THEIR CAREGIVERS, THEIR PARENTS HAVE TO PERFORM TO TRY TO JUST GET BY?

>> SO THE DAY-TO-DAY LIFE OF A FAMILY WITH A CHILD WITH E.B.

IS ONE WHERE PROBABLY IN THE MORNING, YOU KNOW, THE CHILD WAKES UP.

YOU HAVE TO THINK ABOUT SOFT FOODS BECAUSE DIFFICULTY TO EAT FOODS LIKE CHIPS OR ANYTHING HARD WILL TEAR THE INSIDE SKIN OF THEIR MOUTH.

AND THEY HAVE PROBLEMS SWALLOWING.

SO OFTENTIMES THE FOOD HAS TO BE SOFT.

THEN YOU'RE BASICALLY WRAPPING THE SKIN TO PROTECT IT.

SOMETIMES THE WORST EVENT HAPPENS LATER IN THE DAY, IN THE EVENING, AND USUALLY THE KIDS NEED A BATH AND A WOUND DRESSING CHANGE EVERY DAY OR EVERY OTHER DAY, AND IT FEELS LIKE, YOU KNOW, THE PARENT IS TORTURING THE CHILD.

AS A YOUNG CHILD, YOU DON'T KNOW THE CONSEQUENCES, RIGHT.

ALL YOU'RE SAYING TO THE PARENTS IS, I DON'T WANT THIS.

I DON'T WANT TO TAKE THE BANDAGES OFF.

IT HURTS.

AND SO THERE IS A LOT OF NEGOTIATION AND, YOU KNOW, AS A PARENT I JUST THINK ABOUT THAT KIND OF IT DAILY STRUGGLE AND ORDEAL AND THE AMOUNT OF STRENGTH AND CREATIVITY THAT A FAMILY MUST HAVE TO BE ABLE TO BASICALLY WALK THIS DIFFICULT JOURNEY EVERY SINGLE DAY OF THEIR LIFE.

>> MICHAEL HUND, THE APPROACH THAT YOU'RE TAKING IS A VENTURE PHILANTHROPY MODEL.

FOR THE AUDIENCE, EXPLAIN WHAT THAT IS, WHAT THE BENEFITS ARE ZPARD TO A TRADITIONAL MODEL AND WHETHER THAT HAS BEEN THE SORT OF KEY TO UNLOCKING THESE THREE DIFFERENT FDA APPROVALS AND SO FORTH.

>> BOLD MISSIONS REQUIRE INNOVATIVE BUSINESS MODELS.

SO WE'VE ALWAYS BELIEVED THAT WHILE A NONPROFIT ORGANIZATION FOCUSED ON MEDICAL RESEARCH FOR E.B., THAT THE COMMUNITY DESERVES FOR US TO RUN A REALLY GOOD BUSINESS.

AND WHAT THAT LOOKS LIKE FOR US IS VENTURE PHILANTHROPY.

WHAT DOES THAT MEAN?

EVERY PENNY WE INVEST IN RESEARCH AS AN UPSIDE IN WHICH IF THAT SCIENCE AND RESEARCH IS COMMERCIALIZED, A RETURN COMES BACK TO THE FOUNDATION TO REINVEST IN MORE RESEARCH UNTIL ULTIMATELY WE HAVE MORE TREATMENTS AND A CURE.

REALLY THIS CREATES A SUSTAINABLE FUNDRAISING MODEL.

SOMEBODY GIVES US A DOLLAR, WE TURN THAT INTO $6.

IT ALSO SOLVES THE MARKET PROBLEM THAT IS NOT COMPLETELY UNIQUE TO RARE DISEASE WHAT WE CALL THE VALLEY OF DEATH IN MEDICAL RESEARCH.

MOST SCIENCE STRUGGLES TO GET OUT OF A BRILLIANT LAB LIKE DR.

TANG'S IN A BIOTECH OR PHARMACEUTICAL COMPANY TO GO THROUGH THE PHASES OF CLINICAL TRIAL AND GET REGULATORY APPROVALS.

IT CAN HELP MANY OTHER RARE DISEASES AS A MODEL THAT CAN BE SCALED.

>> MICHAEL, THIS FILM WAS SO WONDERFUL AND KIND OF OPENING THE AUDIENCE UP TO THE PERSONAL LIVES, EVEN A SMALL GLIMPSE OF THE LIVES OF SOME OF THE INDIVIDUALS THAT ARE AFFECTED BY THIS AND THEIR FAMILIES.

ONE OF THEM WAS DEANNA MOLINARO.

TELL US ABOUT DEANNA.

SHE TRAGICALLY PASSED AT 31 YEARS OF AGE WHILE MOST PEOPLE WITH E.B.

MIGHT DIE FAR SOONER, RIGHT.

WHAT MOVED YOU ABOUT HER, AND THE TIME THAT YOU KNEW HER?

>> DEANNA WAS A FORCE, A GIANT, A HERO.

YOU HEAR IN THE FILM PRETTY PROFOUND THINGS THAT DEANNA SAYS.

ONE OF THEM THAT STICKS WITH ME, SHE SAYS I LEARNED THAT LIFE IS ABOUT THE QUALITY, NOT THE QUANTITY OF THE YEARS.

AND THE ATTITUDE AND MENTALITY THAT SHE BROUGHT TO HER LIFE EVERY DAY DESPITE THE CHALLENGES THAT SHE FACED, DESPITE THE BURDEN THAT E.B.

PLACED ON HER, SHE GREETED EACH DAY WITH POSITIVITY, WITH JOY, WITH FIRE IN HER BELLY, WITH ART, WITH MUSIC, AND, YOU KNOW, IF YOU GET AN OPPORTUNITY, LOOK AT DEANNA'S ART BECAUSE IT REMINDS US OF WHO SHE IS.

LISTEN TO HER WORDS.

ONE OF THE MOST POWERFUL SCENES IN THE FILM TO ME IS WHEN SHE IS SIT BEING ROWAN DOING MAKEUP TOGETHER.

IT'S THIS MOMENT OF YOU HAVE TWO YOUNG WOMEN THAT WERE DEALT THE SAME GENE, YOU KNOW, AT BIRTH, AND DEANNA KNOWS THAT ROWAN'S LIFE WILL BE IMPROVED BECAUSE OF THE WORK OF PEOPLE LIKE DR.

JEAN TANG, BECAUSE OF THE WORK THAT OUR FOUNDATION HAS BEEN ABLE TO INVEST IN ACROSS THE PLANET.

BUT SHE ALSO KNEW HOW IMPORTANT HER TIME WITH ROWAN WAS TO REALLY BE THAT MENTOR, TO BE THAT FRIEND.

>> DR.

TANG, WITH THE HELP OF THIS PROGRAM, THIS PARTNERSHIP, YOU HAVE BEEN ABLE TO SEE IN THIS PARTICULAR CASE THREE FDA APPROVED TREATMENTS.

IT'S NOT QUITE A CURE, BUT WHAT DO THESE TREATMENTS DO FOR THE DIFFERENT KINDS OF E.B.

THAT EXISTS?

>> RIGHT.

SO ALL OF THESE TREATMENTS WOULDN'T HAVE HAPPENED WITHOUT THE PATIENT FOUNDATION.

SO E.B.

IS ONE OF THE WORST DISEASES YOU'LL EVER HEAR ABOUT.

BUT WE ARE FORTUNATE BECAUSE WE HAVE THE POWER OF, YOU KNOW, WONDERFUL PATRON SAINTS LIKE EDDIE VEDDER AND THE FOUNDATION TO FUND THIS KIND OF RESEARCH.

THERE IS ABSOLUTELY NO WAY THAT FEDERAL GOVERNMENT MONEY AND GRANTS WOULD HAVE BROUGHT US HERE.

SO IN TERMS OF THE FDA APPROVED TREATMENTS, WHAT ARE THEY?

THESE ARE TOPICAL TREATMENTS THAT TRY TO HEAL EXISTING WOUNDS, CLOSE THEM, HEAL THEM SO THE CHILD FEELS LESS PAIN, LESS ITCH.

THEY ARE DEFINITELY HELPING THE PATIENT, THEIR FDA APPROVED.

THE ONE THAT I WORKED AND AT STANFORD IS ONE WHERE WE BIOPSY THE PATIENT'S SKIN CELLS, USE A VIRUS AND PUT IN THE CORRECT GENE AND SEW ON THE GENETICALLY CORRECTED CELLS TO HEAL THEIR SKINS.

THESE MEDICINES ARE COSTLY BECAUSE THERE AREN'T A LOT OF PATIENTS.

AS SCIENTISTS WE ARE TRYING TO THINK HOW TO MAKE THIS SCALABLE, DELIVER A TECHNOLOGY AND MEDICINE THAT CAN BE GIVEN TO PATIENTS WHEREVER THEY LIVE AND AT A PRICE WHERE IT'S NOT GOING TO BANKRUPT INSURANCE.

THESE ARE ALL HARD QUESTIONS.

>> WHAT YOU ARE DESCRIBING IS ALMOST A LAYER OF PRECISION IN MEDICINE THAT I HAVEN'T HEARD OF AND SEEN BEFORE.

IS THIS ENABLED BY CRISPER TECHNOLOGY OR AGAIN HOW DO YOU DO THIS FOR 100 OR 10,000 PATIENTS WITH, YOU KNOW, TO MAKE SURE THAT THE MED SEN WORKS FOR EACH OF THEM?

>> IT'S A GREAT QUESTION.

SO THE FDA APPROVED DRUGS USE VIRUSES CURRENTLY TO INSERT THE ENTIRE COLLAGEN 7 GENE INTO THE PATIENT'S CELLS.

SO NOW THE PATIENT'S CELLS ARE ABLE TO BIND TO THE BOTTOM OF THE SKIN LAYER AND THE WOUNDS WILL HEAL.

THAT PROCESS IS CUMBERSOME.

IT TAKES A LONG TIME.

NOW, WITH CRISPR GENE-EDITING, ONE CHALLENGE IS MANY OF THESE PATIENTS HAVE DIFFERENT MUTATIONS.

SO I BELIEVE NPR REPORTED THIS AND OTHERS.

BUT FDA CREATED A NEW MECHANISM AND GUIDANCE CALLED THE PLAUSIBLE MECHANISM.

THE IDEA WITH CRISPR GENE EDITING, I HAVE THE REAGENTS TO CORRECT PATIENT ONE, MUTATION ONE.

MOST OF THE REAGENTS ARE GOING TO BE THE SAME BETWEEN PATIENT ONE AND PATIENT TWO.

AND SO MAYBE THE REGULATORY BURDEN, THE TOXICOLOGY STUDIES, THE EXPERIMENTS THAT ONE HAS TO DO TO PUT THIS IN HUMANS FOR MUTATION TWO, MUTATION THREE, MUTATION FOUR IS GOING TO BE EASIER AND QUICKER.

AND THAT IS A SPECIFIC WAY IN WHICH GENE-EDITING MEDICINE, PRECISION MEDICINE CAN BE SCALED FASTER AND CHEAPER.

>> DR.

TANG, JUST NOR CLARIFICATION, WHAT IS THE THRESHOLD FOR A DEFINITION OF A RARE DISEASE?

I KNOW AT THE END OF THIS MONTH, FEBRUARY 28th IS GLOBAL RARE DISEASE DAY.

WHAT DO WE MEAN WHEN DE SAY SCHA.

>> THE GOVERNMENT'S DEFINITION OF WHAT QUAL TIES AS A RARE DISEASE IS A DISEASE WHERE THERE IS LESS THAN 200,000 PEOPLE IN THE WORLD.

AND SO IF YOU ADD UP ALL OF THE DIFFERENT TYPES OF RARE DISEASES, IT'S NOT SO RARE.

BUT THE INDIVIDUAL MUTATIONS SUCH AS E.B., IT IS RARE.

AND I'M SO GLAD THAT THERE IS A RARE DISEASE DAY JUST TO RAISE AWARENESS AND TELL PEOPLE MORE ABOUT THE PATIENTS, THE FAMILIES, THEIR NEED.

BUT I FEEL LIKE THIS DOCUMENTARY AND ALL OF THE PEOPLE THAT ARE PART OF IT, I MEAN, THEY DESERVE AN OLYMPIC MEDAL BECAUSE THE ARTISTS, THE MUSICIANS, CREATIVE FILMMAKERS, THE FUNDRAISERS, THE PATIENTS' FAMILIES, I MEAN, IT'S A STORY OF HOPE THAT WILL BRING SCIENCE AND SUCCESS OUT TO THE PUBLIC AND LEAVE IT TO THE ARTISTS AND THE CREATIVE PEOPLE TO TELL THE HUMANITARIAN STORY THAT SOMETIMES SCIENTISTS LIKE MYSELF AREN'T QUITE ABLE TO CONVEY.

>> WHEN YOU LOOK AT DISEASES, IT SEEMS THAT IF THERE IS NOT A CELEBRITY ATTACHED, IT'S ALMOST LIKE A "HUNGER GAMES" OR SURVIVOR COMPETITION WHERE YOU HAVE THIS FINITE RESOURCE AND YOU HAVE ALL THESE RARE DISEASES AND THESE FAMILIES KIND OF COMPETING FOR IT.

AND I DON'T KNOW, YOU KNOW, YOU HAVE BEEN IN THIS SPACE FOR A WHILE, AND I HATE TO BE CYNICAL ABOUT IT IN THAT WAY, BUT IT SEEMS LIKE THAT'S NOT EVERY DISEASE IS KIND OF GETTING ITS DAY IN THE SUN.

>> I DO THINK ONE OF THE SOLUTIONS FOR RARE DISEASE IS AS MUCH AS WE CAN GO IT TOGETHER BECAUSE, AGAIN, IN ISOLATION, THESE DISEASES ARE EXACTLY THAT.

THEY ARE RARE.

BUT WHEN YOU LOOK AT THE COHORT AND AGGREGATE OF ALL OF THOSE LIVING WITH A RARE DISEASE, I MEAN, THAT'S ONE IN TEN PEOPLE ON THE PLANET AFFECTED BY A RARE DISEASE, MORE THAN KANSAS AND HIV COMBINED.

THIS IS A BIG COHORT OF THOSE LIVING WITH RARE DISEASE, SOMETHING WE FOCUS ON AND ARE INTERESTED IN AS, YES, EVERY RARE DISEASE MAY NOT HAVE A CELEBRITY AMBASSADOR.

HOWEVER MOST RARE DISEASES FACE THE SAME CHALLENGES.

FUNDING FOR RESEARCH, RECRUITING FOR CLINICAL TRIALS, HAVING AWARENESS, HAVING A VOICE, THINGS LIKE A RARE DISEASE DAY HELP.

BUT FOR US HOW CAN WE TAKE A MODEL THAT WORKS, THAT'S CERTAINLY BENEFITTED FROM PEOPLE LIKE JILL AND EDDIE VEDDER.

HOW CAN WE SHARE THAT, COME TOGETHER AS A COMMUNITY AND UNITE AS A RARE DISEASE COMMUNITY AND SHARE THOSE SUCCESSES AND BENEFITS FROM FUNDRAISING TO MODELS TO RESEARCH TO SCALABLE NOW TECHNOLOGIES THAT WE HAVE IN MEDICINE AS YOU HEAR DR.

TANG TALK ABOUT.

GENE-EDITING.

THESE ARE THINGS THAT ARE TRANSFERABLE AND SCALABLE.

>> DR.

TANG, SIMILAR QUESTION TO YOU.

IS THERE A METHOD OR A PROCESS THAT YOU CAN SEE GOING FROM E.B.

TO OTHER RARE DISEASES AND WHERE YOU CAN INFORM OTHER RESEARCHERS, SAY HERE IS WHAT I DID, HERE IS WHAT IS PUBLISHED, WHAT YOU CAN RE-CREATE?

>> ABSOLUTELY.

THE GENE-EDITING FIELD IS INCREDIBLE.

AND I KNOW JENNIFER WAS ON YOUR SHOW AND EXPLAINED CRISPR GENE-EDITING AND DISCOVERED IT.

THE CHALLENGE IS IT'S WORKING IN THE LAB.

IN TERMS OF DRUG APPROVALS AND DRUGS GETTING TO THE PATIENTS, IT'S VERY FEW.

SO ANY GENE-EDITING PROGRAM, ANY CLINICAL TRIAL WITH REGULATORY INTERACTIONS WITH THE FDA, THAT WILL SET AN IMPORTANT PRECEDENCE FOR ALL OF THE OTHER GENE-EDITING DRUGS AND WHATEVER DIFFERENT DISEASES.

ANY PROGRESS, ANY RULES, ANY PRECEDENTS YOU SET WITH THE FDA WILL HELP ALL OF THE PROGRAMS.

WHAT IS THE QUALITY OF THE REAGENT, OF THE RNA, HOW MANY PATIENTS DOES IT TAKE TO SEE AN FDA APPROVAL, HOW LONG DO WE HAVE TO FOLLOW THE PATIENTS FOR SAFETY, RIGHT.

SO THE MORE WE'RE ABLE TO PROGRESS IN E.B., I KNOW IT WILL HELP ALL THE OTHER RARE GENETIC DISEASES.

>> DR.

TANG, RECENTLY THE ADMINISTRATION HAD CUT BACK ON ABOUT $9 BILLION WORTH OF GRANTS FOR THE NATIONAL INSTITUTES OF HEALTH.

THAT WAS ABOUT 2,600 DIFFERENT RESEARCH GRANTS, I THINK.

AND I DON'T KNOW IF DIRECTLY E.B.

WAS AFFECTED.

BUT I WONDER HOW HAS THIS END OF FUNDING AFFECTED THE LANDSCAPE OF THE RESEARCH THAT YOU AND YOUR COLLEAGUES ARE DOING INTO RARE DISEASES?

>> ALL I CAN SAY IS THE CUTS ARE TERRIBLE AND THE LANDSCAPE HAS CHANGED.

WE ARE HAVING A HARD ENOUGH TIME CONVINCING AMERICANS TO GO INTO SCIENCE.

AND WITH THAT KIND OF FUNDING CUT AND DESPERATION FOR RESEARCH DOLLARS, IT BASICALLY IS A DARK CLOUD ALL OVER SCIENCE.

IN TERMS OF THE GENE-EDITING TECHNOLOGY, THAT WAS DIRECTLY FUNDED BY THE NIH.

SO THINK ABOUT FOR THE NEXT, YOU KNOW, THREE TO FOUR YEARS WITH THESE CUTS IN SCIENCE FUNDING, WHAT OTHER RESEARCH TECHNOLOGIES ARE WE POTENTIALLY MISSING ON THAT ARE GAME BREAKING?

WHO ARE THE YOUNG SCIENTISTS AND THE LABS THAT WE AREN'T ABLE TO SUPPORT?

AND AS I SAID BEFORE, THERE IS NO WAY THAT THESE FDA APPROVED DRUGS FOR E.B.

WOULD HAVE HAPPENED WITHOUT THE E.B.

PATIENT FOUNDATIONS.

WE DIDN'T HAVE ENOUGH NIH DOLLARS, AND THE PATIENTS AND THE FAMILIES SOLD COOKIES, RAN MARATHONS, DID EVERYTHING THEY CAN TO ASK PEOPLE TO PUT MONEY IN THE JAR FOR US.

SO THAT CAN'T BE REPLICATED FOR EVERY PATIENT DISEASE OR PROBLEM.

AND THAT IS THE ROLE OF THE GOVERNMENT.

I'M SORRY GET ON MY HIGH HORSE, BUT WE NEED TO DO BETTER AS AMERICANS.

>> MICHAEL HUND AND DR.

JEAN TANG, THANK YOU FOR JOINING US.

>> THANK YOU, HARI.

>> THANK YOU.