Rare Diseases Research: Clinical Trial for Niemann-Pick Type C

  Рет қаралды 37,757

Albert Einstein College of Medicine

Albert Einstein College of Medicine

10 жыл бұрын

www.einstein.yu.edu - Determined to find a treatment for children with the degenerative brain disease Niemann-Pick Type C, Steven Walkley, D.V.M., Ph.D., turned a serendipitous laboratory discovery into a successful national research collaboration with other academic institutions and the National Center for Advancing Translational Sciences' program for rare diseases (Therapeutics for Rare and Neglected Diseases). These efforts led to an NIH Phase 1 clinical trial testing cyclodextrin
as a therapy for children with this disease. Dr. Walkley is a professor in the Dominick P. Purpura Department of Neuroscience and director of the Rose F. Kennedy Intellectual and Developmental Disabilities Research Center at Albert Einstein College of Medicine.

Пікірлер: 30
@marcierichie5644
@marcierichie5644
I had never heard of Nieman Pick before watching a Turkish tv series, Kizim, which was based on a Korean series, My Fair Lady. I was shocked to learn of this devastating condition. Thanks to all the dedicated people working on changing the outcome for all the affected children and their families.
@bobbytra1
@bobbytra1 10 жыл бұрын
Amazing people doing amazing things. As a good friend of one family involved in these trials and this work, I just want to say how proud I am of you people. Go from strength to strength.
@drzoranostojic5155
@drzoranostojic5155
Great doctor and great man - Dr Dominic Purpura!!!
@quaziloko
@quaziloko 9 жыл бұрын
My sister had this. Took years to diagnose. Very sad to not know what's going on. Hope anyone with a family member finds out sooner so they get the support they need.
@elnabjelland-hughes8172
@elnabjelland-hughes8172 6 жыл бұрын
Cyclodextrin does work to parents started this whole business with the cyclodextrin their name is Mr. and Mrs. Hemphill they have two daughters who are getting this treatment and have been getting this treatment long before this study was done and they’re doing well
@asifhussain2555
@asifhussain2555 Жыл бұрын
My two kids 1 ur old and 3 yrs old diagnosed with NPC , o don’t know what to do . Some doctors are suggesting liver transplant
@chinnakarri
@chinnakarri Жыл бұрын
My baby is Niemann pick type c any help please 🙏
@captainrahil2446
@captainrahil2446 4 жыл бұрын
My son also surviving with Nieman pick dieses AB please guide any treatment is there ?
@salehahazara5903
@salehahazara5903 Жыл бұрын
Unfortunately my baby have Npc
@rjhenderson2031
@rjhenderson2031 4 жыл бұрын
Are you sure his body isn't just react to muslt
@salmankarimpour8561
@salmankarimpour8561 8 жыл бұрын
Does anyone know if there is a certain treatment for it? my niece got this disorder and we still dont hear a proper response
@katekleinsteuber7652
@katekleinsteuber7652 5 жыл бұрын
What a terrible disease.
@trishstevenson3022
@trishstevenson3022 3 жыл бұрын
Stop using animals
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