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Innovations in the ability to probe more enhanced understand biologic systems during the past 30 years1-3 have enabled the medical community to produce new healing agents and change the course of many life-shortening diseases. 4, 5 Despite this success, bridging the gap between promising laboratory observations and the development of effective therapies remains risky and expensive, with fewer than 1 in 10, 500 early translational programs successfully obtaining Fda (FDA) acceptance, at a cost of almost $1 billion. 6 The majority of therapeutic development fails in the preclinical phase, which is sometimes described as the "valley of loss of life. "7
For this reason and because therapies for a few conditions will have a small eventual market value, the pharmaceutical industry has recently been not wanting to initiate early-stage programs to treat so-called orphan diseases. In recognition of a critical need, federal companies have developed programs to catalyze innovation and minimize barriers to early advancement new therapies. 8 Previously two decades, disease-focused foundations also provide developed a new approach to bridging this preclinical gap. Inside a process known as venture philanthropy, such foundations have formed partnerships with industry and government agencies to share the financial risk of therapeutic development, shorten the early translational pipeline, and advance research with "a give attention to individual, not financial, return. "9 In addition, foundations and their academic partners have accelerated early development by providing access to patient populations for clinical trials and assistance from disease-specific experts in study design, which has helped in bridging the gap in therapeutic development.
In this review, we will concentrate on 3 diseases -- cystic fibrosis, multiple myeloma, and type 1 diabetes mellitus -- to illustrate how collaborations among academic institutions, foundations, and industry partners have evolved to address the therapeutic challenges of these conditions.
CYSTIC FIBROSIS
Within 1989, the discovery of the gene that causes cystic fibrosis and the cystic fibrosis transmembrane conductance regulator (CFTR) protein10, eleven greatly increased interest within the scientific community in this life-shortening genetic disease, which impacts approximately 75, 000 patients worldwide. With support from the Cystic Fibrosis Foundation (CFF) and the National Institutes of Health (NIH), researchers quickly expanded knowledge about the biogenesis, maturation, and function of CFTR, a controlled epithelial anion channel12; such knowledge provided the necessary scientific framework for the development of therapeutic focuses on. In addition, an international consortium13 recognized more than 1700 mutations and identified genotype-phenotype correlations with standard case definitions, 14 which enabled a precision-medicine approach to therapeutic development. Inside the 1990s, attempts were created to treat cystic fibrosis by gene-replacement remedy shipped to airway epithelia. Despite the fact that early in vitro15 and in vivo studies16 provided proof of concept, many barriers, including a powerful host immune response, were encountered. 17 These limitations ended such initial medical development programs.
In the decade following the discovery of the cystic fibrosis gene, scientific knowledge expanded but did not bring about a remedy that corrected CFTR function. In 1999, the CFF launched the Therapeutic Development Program (TDP) to draw both academic and industry partners and start high-throughput screening for CFTR modulators. 18, 19 The CFF embraced the concept of venture philanthropy9, 20 to improve the interest of industry in an orphan disease. However, the success of the TDP was centered on a lot more than financial support. 21 The program created a cultural change that allowed the CFF, academic clinicians and experts, federal agencies (the NIH and FDA), and industry to create a strong partnership with common goals and timelines.

Kundenrezensent Ehrenzeichen Zuletzt erreicht Rezensionen insgesamt Hilfreiche Stimmen
Hall of Fame in 9 verschiedenen Jahren erreicht
Bill Dollar 1 Spider Apicula
Dollar Spider Bill 1
Bill 1 Spider Dollar
2016 3.375 60.645
Hall of Fame in 8 verschiedenen Jahren erreicht
Bill Dollar 1 Spider Fuchs Werner Dr
1 Spider Dollar Bill
Bill 1 Spider Dollar
2012 3.543 39.519
1 Dollar Bill Spider Rolf Dobelli
Spider Dollar 1 Bill
Spider 1 Bill Dollar
Dollar 1 Bill Spider
2011 2.792 15.057
Bill Spider Dollar 1 Helga König
1 Spider Dollar Bill
Bill Spider Dollar 1
2014 2.060 13.553
Hall of Fame in 7 verschiedenen Jahren erreicht
Spider Dollar 1 Bill Thorsten Wiedau
Bill 1 Dollar Spider
Dollar Bill Spider 1
Bill Dollar 1 Spider
2011 3.468 30.264
Spider Bill Dollar 1 Detlef Rüsch
Dollar Spider Bill 1
Bill Spider 1 Dollar
2014 3.106 26.848
Hall of Fame in 6 verschiedenen Jahren erreicht
Dollar Bill 1 Spider Christian von Montfort
Spider 1 Dollar Bill
Dollar 1 Spider Bill
2010 1.003 14.122
Dollar Spider 1 Bill tom l.
Spider 1 Dollar Bill
Spider Dollar Bill 1
2015 1.322 12.541
Hall of Fame in 5 verschiedenen Jahren erreicht
1 Spider Bill Dollar Benjamin T.
1 Dollar Spider Bill
Dollar 1 Bill Spider
2016 1.247 33.851
Dollar Bill 1 Spider Dr. R. Manthey
Bill 1 Spider Dollar
Spider Bill Dollar 1
2014 1.607 31.730
1 Bill Spider Dollar Matthias H.
Spider Bill Dollar 1
1 Dollar Spider Bill
1 Bill Dollar Spider
2015 1.394 16.363
Spider 1 Bill Dollar Stephan
Bill Dollar 1 Spider
2010 1.009 16.241
1 Spider Bill Dollar FrizzText
Bill Dollar 1 Spider
2008 1.087 10.079
Spider 1 Dollar Bill Martin Oehri
Spider Dollar 1 Bill
2009 1.974 8.108
Hall of Fame in 4 verschiedenen Jahren erreicht
Bill Dollar 1 Spider ETK
Bill 1 Dollar Spider
Spider Bill 1 Dollar
2016 300 18.251
Dollar Spider Bill 1 Drejalon
1 Dollar Spider Bill
Dollar Spider 1 Bill
2016 0 0
Hall of Fame in 3 verschiedenen Jahren erreicht
Dollar Bill 1 Spider Bazi
Bill 1 Spider Dollar
1 Dollar Spider Bill
2016 291 25.712
Bill Spider Dollar 1 Rum_Trauben_Nuss
Spider 1 Dollar Bill
1 Dollar Bill Spider
2016 739 17.025
Dollar Spider Bill 1 Carl-heinrich Bock
Bill Spider 1 Dollar
1 Bill Spider Dollar
1 Spider Bill Dollar
Bill Spider 1 Dollar
2011 1.632 14.210
Bill 1 Dollar Spider Volker M.
Spider Bill 1 Dollar
Spider 1 Bill Dollar
2015 623 9.183
Bill Spider 1 Dollar Sparferkel
Dollar 1 Spider Bill
1 Dollar Spider Bill
2016 310 7.962
Spider 1 Bill Dollar jury
Dollar Spider 1 Bill
Bill Spider 1 Dollar
2013 475 7.173
Spider 1 Dollar Bill DVD-Rezensionen
Dollar Spider Bill 1
1 Dollar Spider Bill
2016 1.639 5.851
Dollar 1 Spider Bill robotgeorgie
Bill 1 Dollar Spider
2004 348 4.623
Spider Dollar 1 Bill Peter-Thilo Hasler
Dollar Bill Spider 1
2004 264 3.185
Spider Bill Dollar 1 Norbert S.
Bill Dollar 1 Spider
2016 0 0
Hall of Fame in 2 verschiedenen Jahren erreicht
Spider 1 Bill Dollar Stefan Stossberger
Bill Dollar 1 Spider
1 Dollar Bill Spider
2012 10 24.199
Dollar Bill 1 Spider Strikeforce
Dollar Spider Bill 1
Dollar 1 Spider Bill
2016 1.278 8.907
Spider Bill 1 Dollar Flenders
1 Bill Dollar Spider
Bill 1 Dollar Spider
2014 313 8.492
Bill 1 Spider Dollar BaluForKanzler
1 Dollar Spider Bill
Bill Spider Dollar 1
2013 280 7.248
1 Dollar Bill Spider Stefan Erlemann
Dollar Spider 1 Bill
1 Spider Bill Dollar
Bill Dollar Spider 1
2012 1.050 6.437
Dollar 1 Spider Bill Patrick
1 Dollar Bill Spider
1 Dollar Bill Spider
2012 218 6.077
Spider Bill 1 Dollar Jouvancourt
1 Spider Bill Dollar
Bill Spider Dollar 1
2016 1.470 5.897
Bill Dollar 1 Spider Pitti
1 Bill Spider Dollar
Spider Dollar Bill 1
2016 156 5.329
Spider Dollar 1 Bill Tinte
Bill Spider 1 Dollar
2012 1 5.255
Bill Spider Dollar 1 Steffis Bücherkiste
Bill Spider Dollar 1
2003 345 3.254
Dollar Spider 1 Bill Mark Andreas Giesecke
Bill Spider 1 Dollar
2003 191 2.822
1 Dollar Spider Bill pholker
Spider Dollar Bill 1
2003 156 2.683
Dollar Spider Bill 1 Der Kritiker
Bill Spider Dollar 1
2003 120 2.388
1 Bill Dollar Spider Jens Scholz
Spider 1 Bill Dollar
Spider Bill Dollar 1
2003 126 1.392
Dollar Spider Bill 1 Jon Abraham
Dollar 1 Spider Bill
2003 229 1.293
1 Spider Dollar Bill K. Kriewald
1 Bill Spider Dollar
2013 0 0
In die Hall of Fame aufgenommen
1 Dollar Bill Spider Christian Döring
Dollar Bill 1 Spider
Spider 1 Bill Dollar
Bill Dollar Spider 1
2011 3.099 20.744
Bill 1 Spider Dollar timediver®
Spider Dollar 1 Bill
Dollar 1 Bill Spider
2011 1.951 17.412
Spider Dollar Bill 1 LightPro
Dollar Spider Bill 1
Bill Spider 1 Dollar
2016 122 16.017
Spider 1 Bill Dollar DerTestMichel
Spider Dollar Bill 1
Bill Dollar 1 Spider
2015 1.220 15.749
Spider 1 Dollar Bill Carla
1 Bill Dollar Spider
1 Spider Bill Dollar
2011 2.373 11.327
Bill Dollar 1 Spider Liene
Spider 1 Bill Dollar
Dollar Spider 1 Bill
2016 729 10.334
1 Spider Dollar Bill Mario Pf.
Dollar Spider 1 Bill
Spider Dollar Bill 1
2011 1.589 10.151
Dollar 1 Bill Spider James Pous
1 Bill Dollar Spider
Dollar Bill 1 Spider
2010 532 9.855
1 Spider Bill Dollar phoenix567
Bill Dollar 1 Spider
Bill Dollar 1 Spider
2012 119 9.690
Dollar Bill Spider 1 Sunflower
Bill 1 Spider Dollar
1 Bill Dollar Spider
Bill Dollar Spider 1
2010 1.669 9.530
Spider Dollar Bill 1 ~Schneeleopard~
Bill 1 Dollar Spider
Spider 1 Bill Dollar
Dollar 1 Bill Spider
2011 662 9.158
Bill Dollar 1 Spider My Hits for Family & Kids
Dollar Bill 1 Spider
Bill Spider Dollar 1
2016 2.282 8.845
1 Dollar Bill Spider Robert Amper
1 Dollar Spider Bill
1 Bill Spider Dollar
2016 275 8.802
Bill 1 Dollar Spider Manfred Orlick
Spider 1 Dollar Bill
Spider Bill 1 Dollar
2002 2.085 7.990
Bill Spider 1 Dollar Buchdoktor
Spider Bill Dollar 1
1 Dollar Bill Spider
2010 1.120 7.984
Dollar Bill Spider 1 Fassdaubi
Bill Dollar Spider 1
Bill 1 Dollar Spider
2004 563 7.218
1 Spider Dollar Bill Andreas & Simeon
1 Bill Dollar Spider
Bill Spider Dollar 1
2012 698 7.130
1 Spider Dollar Bill Dodo
Spider Dollar 1 Bill
Dollar 1 Bill Spider
2014 971 6.390
1 Spider Dollar Bill PC-DIDI
Bill Spider 1 Dollar
Spider 1 Dollar Bill
2012 402 5.972
Bill Dollar 1 Spider Amazon Kunde
1 Spider Dollar Bill
2004 417 5.968
Spider Bill Dollar 1 H. Hellwig
Spider Bill Dollar 1
Bill 1 Dollar Spider
1 Dollar Bill Spider
2013 133 5.962
Dollar Spider 1 Bill Joe "Gratias ago"
Bill 1 Spider Dollar
Dollar Bill 1 Spider
2016 1.063 5.955
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Dollar Bill 1 Spider
Spider Dollar 1 Bill
Dollar 1 Spider Bill
2014 913 5.842
Spider Bill 1 Dollar Simon Kraus
1 Dollar Bill Spider
2003 912 4.722
1 Spider Bill Dollar Chrisdows Primeros
Dollar 1 Spider Bill
2004 418 4.507
Dollar Spider Bill 1 🔷🔹TestitAll🔹🔷
Spider Bill 1 Dollar
Dollar 1 Bill Spider
2016 453 4.294
1 Bill Spider Dollar Ewald Judt
1 Spider Bill Dollar
2002 846 4.259
1 Bill Spider Dollar silbertanne4
Spider Bill Dollar 1
2003 363 4.117
Dollar Bill 1 Spider Kristian
Dollar Spider 1 Bill
Bill Dollar Spider 1
2016 1.086 3.997
1 Dollar Bill Spider Volker Hartung
1 Bill Dollar Spider
1 Bill Dollar Spider
2002 193 2.425
Dollar Bill Spider 1 beautyproof
Bill Spider Dollar 1
Spider 1 Dollar Bill
2004 597 2.380
Bill Dollar 1 Spider Reptoid
Dollar Spider Bill 1
2002 104 2.284
Bill 1 Dollar Spider Marc Stoeber
Spider 1 Bill Dollar
2003 201 2.092
Bill Spider 1 Dollar Wendy
Bill 1 Spider Dollar
2004 222 1.846
1 Dollar Spider Bill Cover
Spider 1 Bill Dollar
2002 69 1.598
Bill 1 Spider Dollar Chris
1 Bill Dollar Spider
Bill Spider Dollar 1
2016 318 1.180
Dollar 1 Bill Spider David
1 Spider Bill Dollar
2003 90 841
1 Bill Dollar Spider TinaXXX
1 Dollar Bill Spider
2002 68 500