. . . you're not alone, p9, in questioning etep's efficacy . . . mr. market gives srpt a market cap of just $1.2 billion -- granted, that's a princely sum for sure, but it's really peanuts for a company that supposedly owns all rights to a DMD drug that's destined to be a clinical slam dunk . . . not sayin' it won't happen, but there's still plenty of risk between here and there . . .
. . . a lifetime of treatment using a marginally effective drug . . . that's what you're hoping for? . . . jeez louise, b9, set your sights a little higher . . . look at the work being done using genetic engineering at Duke University and elsewhere . . . a real cure for DMD may be closer than you think . . . and, yes, I say that owning a half dozen genetic engineering outfits like BLUE . . . good luck with your trade . . .
. . . get back to me, w2, when you actually know what confirmation bias is . . . in the meantime, good luck with your wagers here and elsewhere . . .
. . . I’d recommend you go to the BMRN website, cs, and get the full facts . . . gathering information solely from the like-minded on a rabid message board like this one is to court disaster -- since all you’re likely to get here is more confirmation bias . . . good luck in any case with your stock speculations . . .
. . . June 17, 2015 – “BioMarin Pharmaceutical Inc. today announced positive results of a Phase 2 proof-of-concept and dose finding study of BMN 111 (vosoritide), an analog of C-type Natriuretic Peptide (CNP), in children with achondroplasia. Achondroplasia is the most common form of human dwarfism. Vosoritide has Orphan designation in both the United States and Europe.” . . . BMRN – among the best in the orphan drug business . . . watch and learn . . .
. . . discussed "wildly" is right . . . what I have read is that these cures will likely have fixed pricing with payments spread over a period of years -- so long as the treatment remains efficacious -- until the total cost has been covered . . . for instance, an orphan disease treatment from QURE -- recently approved in Europe -- has a cost of approximately US $1.3 million . . .
. . . "no turning back"? . . . jeez louise, cs . . . if you think for a minute that someone facing a truly debilitating/life-threatening genetic illness would not jump at the chance for a one-shot REAL CURE -- if it has a proven record of success with negligible side effects -- you're drinking too much of the kool aid here . . . why do think PFE, BMY, NVS and other big pharmas have partnered -- worldwide -- with genetic engineering outfits over the past two years? . . .
. . . hats off to blue, indeed, b9 . . . but don't confuse its efforts to genetically engineer one-time, permanent cures for orphan diseases with the chemical therapies now being developed by srpt and bmrn . . . just sayin’ . . .
June 8, 2015: "BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) announced today the submission of a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for drisapersen, an investigational antisense oligonucleotide drug candidate for the treatment of the largest subset of Duchenne muscular dystrophy (DMD) amenable to single exon skipping. . . . In Europe, it is estimated there are 23,000 boys with Duchenne Muscular Dystrophy, and approximately 3,000 of those would be candidates for drisapersen. In BioMarin's commercial territories, approximately 85 percent of Duchenne patients are located outside of the United States, including Western Europe, Middle East, Eastern Europe, Latin America and Japan. Western Europe has the largest patient population among those areas, exceeding the United States by around 30 percent. . . . The company recently submitted a New Drug Application (NDA) to the United States Food and Drug Administration (FDA) for drisapersen in April 2015.” . . .