Posts Tagged ‘Phenylketonuria’

Orphan Drugs Are Good! BioMarin Pharmaceuticals

Orphan Drugs Are Good! BioMarin Pharmaceuticals

Courtesy of Pharmboy

Sick child visited at

A rare disease, sometimes known as an orphan disease, is any disease that is not common. Typically, a rare disease has such a low prevalence in a population that a physician in a busy general practice would not expect to see more than one case a year. Most rare diseases are genetic--present throughout the person’s entire life, even if symptoms do not appear immediately. However, many rare diseases appear early in life, and about 30% of children with rare diseases die before reaching their fifth birthdays.

No single cutoff number has been agreed upon for which a disease is considered rare. A disease may be considered rare in one part of the world, or in a particular group of people, but still be common in another. In the United States of America, the Rare Disease Act of 2002 defines rare disease strictly according to prevalence, as any disease or condition that affects less than 200,000 persons in the United States, or about 1 in 1,500 people.

BioMarin’s (BMRN) core business and research is in enzyme replacement therapies for orphan diseases. They are the first company to provide therapeutics for mucopolysaccharidosis type I (MPS I), by manufacturing Aldurazyme (commercialized by Genzyme Corporation). BioMarin is also the first company to provide therapeutics for Phenylketonuria (PKU)

As of 2005, BioMarin commercialized arylsulfatase B (Naglazyme) as an enzyme replacement therapy for the treatment of mucopolysaccharidosis VI (MPS VI), and in 2007 a drug version of tetrahydrobiopterin (Kuvan), the first medication-based intervention to treat phenylketonuria.

On 11/30/09, BioMarin announced that the FDA has granted orphan drug designation for 3,4-diaminopyridine (3,4-DAP), amifampridine phosphate, for the rare autoimmune disease Lambert Eaton Myasthenic Syndrome (LEMS). 3,4-DAP has previously received orphan drug designation in the E.U. Also, in October 2009, the Committee for Medicinal Products for Human Use of the European Medicines Evaluations Agency adopted a positive opinion recommending approval of amifampridine phosphate for LEMS. If approved by the European Commission, amifampridine phosphate will be the first approved treatment for LEMS, thereby conferring orphan drug protection and providing ten years of market exclusivity in Europe. BMRN expects to meet with the FDA in early 2010 to determine the necessary regulatory path for
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Phil's Favorites

Must the president be a moral leader?

 

Must the president be a moral leader?

President Donald Trump, former President Barack Obama and former President Bill Clinton, during the funeral for former President George H.W. Bush. AP Photo/Alex Brandon, Pool

Courtesy of Michael Blake, University of Washington

The best presidents – including figures such as Abraham Lincoln and George Washington – are celebrated not only as good leaders, but as good men. They...



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Zero Hedge

"This Time Is Not Different" - Saxobank Warns The Temporary Calm Won't Last Long

Courtesy of Zero Hedge

By Christopher DEMBIK, Head of Macroeconomic Analysis, Saxobank

Investors seeking to track the probability of an incoming US recession must familiarise themselves with the yield curve, a key indicator.

Based on the market’s favorite indicator, the yield curve, the risk of US recession is becoming increasingly credible. Hopes for growth improvements may vanish quickly if policymakers don’t step in to stimulate the economy

Negative wealth effect due to lower house prices

Recession probability models for the US have been all over the place of late. Saxo Bank uses the recession probability tracker from the Federal Reserve Bank ...



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Digital Currencies

Cryptos Are Surging: Bitcoin, Ethereum Hit One-Month Highs As Institutions Dip Toes

Courtesy of Zero Hedge

Cryptocurrencies are surging while the US equity markets take the day off. Ethereum is up over 18% from Friday's 'close' and the rest of the crypto space is a sea of green. While no immediate catalyst (headline or technical level) is clear, increasing chatter over institutional investors dipping their toes in the space have prompted an extension of the positive trend.

A sea of green...

Source: Coin360

Ethereum is leading the charge follow...



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Chart School

Weekly Market Recap Feb 17, 2019

Courtesy of Blain.

The “V” shape bounce continues in unrelenting fashion as bulls are stampeding bears in 2019!  All due to a little “patience” from the Federal Reserve.  It is really quite breathtaking but we have seen it repeatedly the past decade as the Federal Reserve pours gas on the market.  Hopes for a deal with China also spurred the action upward.  Rallies (both with gap ups) on Tuesday and Friday provided the juice this week.   The S&P 500 is back over its 200 day moving average after being below for 46 days – it’s longest period of time below that level since March 2016.

Mat Klody, chief investment officer at Keebeck Wealth Management, told MarketWatch that the major benchmarks’ steady march higher since the beginning of the year is being driven &#x...



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ValueWalk

JNJ CEO Alex Gorsky On Partnering With Apple [Full Transcript]

By Jacob Wolinsky. Originally published at ValueWalk.

CNBC Exclusive: CNBC Transcript: JNJ CEO Alex Gorsky Speaks to CNBC’s Jim Cramer Today

Image source: CNBC Video Screenshot

WHEN: Today, Friday, February 15, 2019

WHERE: CNBC’s “Mad Money w/ Jim Cramer

The following is the unofficial transcript of a CNBC EXCLUSIVE interview with JNJ CEO Alex Gorsky and CNBC’s Jim Cramer on CNBC’s “Mad Money w/ Jim Cramer” (M-F 6PM – 7PM) today Friday, February 15. The following is a link to video from th...



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Kimble Charting Solutions

Gasoline bullish breakout could fuel higher prices, says Joe Friday

Courtesy of Chris Kimble.

Are we about to pay much higher prices at the gas pump? Possible!

This chart looks at Gasoline futures over the past 4-years. Gasoline has become much cheaper at the pump, as it fell nearly 50% from the May 2018 highs. The decline took it down to test 2016 & 2017 lows at (1). While testing these lows, Gasoline could be forming a bullish inverse head & shoulders pattern over the past few months.

Joe Friday Just The Facts- If Gasoline breaks out at (2), we could all see higher prices at the gas pump. If a breakout does...



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Insider Scoop

10 Stocks To Watch For February 15, 2019

Courtesy of Benzinga.

Some of the stocks that may grab investor focus today are:

  • Wall Street expects PepsiCo, Inc. (NASDAQ: PEP) to report quarterly earnings at $1.49 per share on revenue of $19.52 billion before the opening bell. PepsiCo shares rose 0.2 percent to $112.82 in after-hours trading.
  • NVIDIA Corporation (NASDAQ: NVDA) reported upbe...


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Biotech

Cancer: new DNA sequencing technique analyses tumours cell by cell to fight disease

Reminder: We are available to chat with Members, comments are found below each post.

 

Cancer: new DNA sequencing technique analyses tumours cell by cell to fight disease

Illustration of acute lymphoblastic leukaemia, showing lymphoblasts in blood. Kateryna Kon/Shutterstock

Courtesy of Alba Rodriguez-Meira, University of Oxford and Adam Mead, University of Oxford

...

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Members' Corner

Why Trump Can't Learn

 

Bill Eddy (lawyer, therapist, author) predicted Trump's chaotic presidency based on his high-conflict personality, which was evident years ago. This post, written in 2017, references a prescient article Bill wrote before Trump even became president, 5 Reasons Trump Can’t Learn. ~ Ilene 

Why Trump Can’t Learn

Donald Trump by Gage Skidmore (...



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Mapping The Market

Trump: "I Won't Be Here" When It Blows Up

By Jean-Luc

Maybe we should simply try him for treason right now:

Trump on Coming Debt Crisis: ‘I Won’t Be Here’ When It Blows Up

The president thinks the balancing of the nation’s books is going to, ultimately, be a future president’s problem.

By Asawin Suebsaeng and Lachlan Markay, Daily Beast

The friction came to a head in early 2017 when senior officials offered Trump charts and graphics laying out the numbers and showing a “hockey stick” spike in the nationa...



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OpTrader

Swing trading portfolio - week of September 11th, 2017

Reminder: OpTrader is available to chat with Members, comments are found below each post.

 

This post is for all our live virtual trade ideas and daily comments. Please click on "comments" below to follow our live discussion. All of our current  trades are listed in the spreadsheet below, with entry price (1/2 in and All in), and exit prices (1/3 out, 2/3 out, and All out).

We also indicate our stop, which is most of the time the "5 day moving average". All trades, unless indicated, are front-month ATM options. 

Please feel free to participate in the discussion and ask any questions you might have about this virtual portfolio, by clicking on the "comments" link right below.

To learn more about the swing trading virtual portfolio (strategy, performance, FAQ, etc.), please click here ...



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Promotions

Free eBook - "My Top Strategies for 2017"

 

 

Here's a free ebook for you to check out! 

Phil has a chapter in a newly-released eBook that we think you’ll enjoy.

In My Top Strategies for 2017, Phil's chapter is Secret Santa’s Inflation Hedges for 2017.

This chapter isn’t about risk or leverage. Phil present a few smart, practical ideas you can use as a hedge against inflation as well as hedging strategies designed to assist you in staying ahead of the markets.

Some other great content in this free eBook includes:

 

·       How 2017 Will Affect Oil, the US Dollar and the European Union

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About Phil:

Philip R. Davis is a founder Phil's Stock World, a stock and options trading site that teaches the art of options trading to newcomers and devises advanced strategies for expert traders...

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