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In addition to its clinical trials, Prosensa were running a
Natural History Study, which has completed enrollment with 269 patients across the US, Europe and Latin America. The purpose of this study was to characterize the natural history and progression of DMD, to help inform the design of future
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Prosensa commenced operations in 2002 and is located in Leiden, The
Netherlands. The company works closely together with academia, patient groups and experts worldwide. Prosensa entered into an exclusive licensing agreement in 2003 with Leiden University Medical Center (LUMC) for their proprietary
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were in advanced preclinical development. In parallel, Prosensa also had an advanced program called PROSPECT into pre-clinical testing, which included a new and innovative application of its exon-skipping technology platform, applying multiple exon skipping, to specifically target rarer mutations
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An
American shareholder filed a class action against Prosensa in New York court in 2014, alleging that he felt misled in the June 2013 NASDAQ IPO. His assumption was, that Prosensa would have developed a drug against Duchenne disease and would receive royalties for it in the short term. However,
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was a biotechnology company engaged in the discovery, development and commercialization of RNA-modulating therapeutics. The company targets genetic disorders with a large unmet medical need, with a primary focus on neuromuscular and neurodegenerative disorders such as
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279:"BioMarin and Prosensa Holding N.V. Reach Agreement on Intended Public Offer for 100% of Prosensa's Outstanding Stock; Will Add Duchenne Muscular Dystrophy Products to Rare-Disease Portfolio"
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In May 2016, BioMarin discontinued the clinical and regulatory development of drisapersen, as well as phase 2 studies of BMN 044, BMN 045 and BMN 053 (formerly PRO044, PRO045 and PRO053).
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studies, to capture biomarkers of safety and disease progression and to provide comparative data for the development of rare exons for which formal controlled trials are not feasible.
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technology to develop treatments for DMD, other neuromuscular disorders and indications outside the field of neuromuscular disorders.
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three months later it would have been revealed that the drug did not really work, resulting in a sharp drop in its share price.
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Prosensa's portfolio of clinical and pre-clinical RNA-based drug candidates is currently focused on the treatment of DMD.
411:"BioMarin Announces Withdrawal of Market Authorization Application for Kyndrisa™ (drisapersen) in Europe (NASDAQ:BMRN)"
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gene. This approach could have applicability between 5-13% of the DMD population.
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In
January 2016, the FDA rejected drisapersen, largely on the basis of toxicity.
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381:"FDA rejects BioMarin's muscle wasting drug; Sarepta drug in focus"
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Leidse biofarmaceut in VS voor rechter na kelderende beurskoers
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199:, has completed Phase III clinical trials and (in June 2015) a
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agreed to pay up to $ 840 million to acquire
Prosensa.
351:"BioMarin to Pay up to $ 840 Million for Prosensa"
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491:Biotechnology companies disestablished in 2014
301:"FDA Accepts BioMarin's NDA for Drisapersen"
486:Biotechnology companies established in 2002
454:Historical business data for Prosensa N.V.:
231:(initially in exon 10 to 30 region) in the
476:Biotechnology companies of the Netherlands
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481:Companies formerly listed on the Nasdaq
307:from the original on September 30, 2015
222:were also in clinical development, and
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391:from the original on January 12, 2017
361:from the original on 27 November 2014
176:. Prosensa was acquired by BioMarin.
303:. Parent Project MD. June 29, 2015.
516:Dutch companies established in 2002
349:Walker, Joseph (24 November 2014).
285:. November 24, 2014. Archived from
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511:Companies disestablished in 2015
203:(NDA) has been accepted by the
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506:2013 initial public offerings
501:2015 mergers and acquisitions
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254:Related events since the sale
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210:Three additional products,
166:Duchenne muscular dystrophy
16:Dutch biotechnology company
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195:Prosensa's lead product,
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248:BioMarin Pharmaceutical
180:History up to the sale
20:Prosensa Therapeutics
387:. January 14, 2016.
201:New Drug Application
174:Huntington's disease
355:Wall Street Journal
142:Number of employees
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415:investors.bmrn.com
331:2016-03-05 at the
289:on March 22, 2015.
246:In November 2014,
170:myotonic dystrophy
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103:, Giles Champion
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445:Official website
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154:www.prosensa.com
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185:RNA modulation
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135:Pharmaceuticals
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437:External links
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421:on 2016-06-04
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54:Biotechnology
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419:the original
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393:. Retrieved
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68:Headquarters
25:Company type
458:SEC filings
395:January 17,
197:drisapersen
119:David Mott
76:Netherlands
470:Categories
425:2016-06-09
311:August 28,
265:References
233:dystrophin
83:Key people
45:: RNA
146:89 (2014)
37:Traded as
389:Archived
365:14 March
359:Archived
329:Archived
305:Archived
283:BioMarin
161:Prosensa
131:Products
123:Chairman
50:Industry
168:(DMD),
151:Website
60:Founded
228:PRO055
224:PRO052
220:PRO053
216:PRO045
212:PRO044
205:US FDA
172:, and
72:Leiden
43:Nasdaq
29:Public
397:2017
367:2017
313:2015
226:and
218:and
63:2002
115:CBO
107:CMO
99:CFO
91:CEO
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