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

Move Over, Millennials

 

Move Over, Millennials

Courtesy of 

Not all young people share the same daily habits or the same views of the world.

Millennials are cutting the cord. Gen Z doesn’t know what the cord is. 45% of Millennials still watch broadcast tv, compared with 26% of 16-17 year olds.

Millennials play fantasy sports. Gen Z watches eSports. Only 18% of 18-24 year olds watch traditional sports, compared with 30% of Millennials.

...

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Zero Hedge

Pennsylvania Governor Bans Alcohol Sales On The Day Before Thanksgiving

Courtesy of ZeroHedge View original post here.

Democratic Pennsylvania Governor Tom Wolf is tapping into his state's Quaker roots to deliver an economy-sized dose of Thanksgiving disappointment. In an effort to avert a coronavirus-inspired lockdown, the governor said Monday that he would ban alcohol sales in the state on the day before Thanksgiving via executive order.

Bars and restaurants should stop selling alcohol starting at 1700ET on Wednesday until 0800ET Thanksgiving morning. Since Thanksgiving is typic...



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Politics

Profiles in Cowardice

 

Profiles in Cowardice

Most elected Republicans in Washington are failing the test by refusing to stand up to Trump. Their cowardice is one of the worst betrayals of public trust in the history of our republic.

By Robert Reich writing at Common Dreams

American democracy will continue to be endangered by House and Senate Republicans who lack the moral courage to do what’s right. (Photo: Alex Wong/Getty Images)

Financial r...



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ValueWalk

AstraZeneca Shares Slide After Weak Covid Vaccine Results

By Gorilla Trades. Originally published at ValueWalk.

Commenting on AstraZeneca’s Covid vaccine results and today’s trading, Gorilla Trades strategist Ken Berman said:

Q3 2020 hedge fund letters, conferences and more

AstraZeneca Shares Fall After Annoucing Weak Results Of Its Covid Vaccine

The major indices are mixed and flat at midday despite a positive start to the session on Wall Street. The Nasdaq’s relative weakness has been weighing on the broader market in early trading, following yet another promising ...



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Kimble Charting Solutions

Transports Sending Strong Bullish Message To Other Dow Indices?

Courtesy of Chris Kimble

Are Transportation stocks about to send a quality bullish message to other Dow indices this month? Sure could be!

This 3-pack looks at the Dow Jones Industrials, Transports, and Utilities indices on a monthly basis.

One week from the end of a month, the DJ Transports are attempting an important bullish breakout at (1). Unless a sharp reversal takes place in the next week, Transports could close out the month at new monthly closing highs!

The Dow is attempting to close at all-time highs this month, while the Dow Utilities Index remains a few percent below 2020 highs....



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Biotech/COVID-19

COVID-19 vaccines were developed in record time - but are these game-changers safe?

 

COVID-19 vaccines were developed in record time – but are these game-changers safe?

The pandemic rages as the world waits for COVID-19 vaccines. Peter Zelei Images/Getty Images

Courtesy of William Petri, University of Virginia

There are now two COVID-19 vaccines that, at least according to preliminary reports, appear to be 94.5% and ...



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Digital Currencies

Dalio Admits "I Might Be Missing Something" As Bitcoin Surges Above $18,000

Courtesy of ZeroHedge

Since the US election, Bitcoin prices (in USD) have surged a stunning 40%, also lurching higher after each vaccine headline hit.

Source: Bloomberg

Getting ever closer to its all-time record high...

Source: Bloomberg

As crypto prices soared overnight, Bridgewater Associates founder Ray Dalio stepped back into the fray, saying in a Twitter thread that “I might be missing something about Bitco...



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Chart School

Inflation Deflation Cycle Review

Courtesy of Read the Ticker

Structural change forces the current narrative to change. One such structural change would be a period elevated inflation.

Inflation is a condition which effects many economic models, we only have to review the history books of the 1970's to see how asset classes reacted then and how they may react in the future. 

In periods of inflation there are short periods of deflation, as markets adjust from one theme to another it usually does so with some pain. The short answer: Nothing moves in a straight line and high volatility will shock the most experience investor.  

Long term cycles raise the question, if happened before is it likely to happen again. Price does not always fit the sine wave cycle neatly, but if the cycle is tru...

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Mapping The Market

COVID-19 Forces More Than Half of Asset Management Firms to Accelerate Adoption of Digital Marketing Technology

By Jacob Wolinsky. Originally published at ValueWalk.

There is no doubt that the use of technology to support client engagement initiatives brings both opportunities and threats but this has been brought into sharp focus this year with the COVID-19 pandemic.

The crisis has brought to the fore the need for firms to enable flexibility in client engagement – the expectation that providers will communicate to clients on their terms, at their speed and frequency and on their preferred channels, is now a given. This is even more critical when clients are experiencing unparalleled anxiety from both market conditions and their own personal circumstances.

...

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The Technical Traders

Adaptive Fibonacci Price Modeling System Suggests Market Peak May Be Near

Courtesy of Technical Traders

Our Adaptive Fibonacci Price Modeling system is suggesting a moderate price peak may be already setting up in the NASDAQ while the Dow Jones, S&P500, and Transportation Index continue to rally beyond the projected Fibonacci Price Expansion Levels.  This indicates that capital may be shifting away from the already lofty Technology sector and into Basic Materials, Financials, Energy, Consumer Staples, Utilities, as well as other sectors.

This type of a structural market shift indicates a move away from speculation and towards Blue Chip returns. It suggests traders and investors are expecting the US consumer to come back strong (or at least hold up the market at...



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Lee's Free Thinking

Texas, Florida, Arizona, Georgia - The Branch COVIDIANS Are Still Burning Down the House

 

Texas, Florida, Arizona, Georgia – The Branch COVIDIANS Are Still Burning Down the House

Courtesy of Lee Adler, WallStreetExaminer 

The numbers of new cases in some of the hardest hit COVID19 states have started to plateau, or even decline, over the past few days. A few pundits have noted it and concluded that it was a hopeful sign. 

Is it real or is something else going on? Like a restriction in the numbers of tests, or simply the inability to test enough, or are some people simply giving up on getting tested? Because as we all know from our dear leader, the less testing, the less...



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Insider Scoop

Economic Data Scheduled For Friday

Courtesy of Benzinga

  • Data on nonfarm payrolls and unemployment rate for March will be released at 8:30 a.m. ET.
  • US Services Purchasing Managers' Index for March is scheduled for release at 9:45 a.m. ET.
  • The ISM's non-manufacturing index for March will be released at 10:00 a.m. ET.
  • The Baker Hughes North American rig count report for the latest week is scheduled for release at 1:00 p.m. ET.
...

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Feb. 26, 1pm EST

Click HERE to join the PSW weekly webinar at 1 pm EST.

Phil will discuss positions, COVID-19, market volatility -- the selloff -- and more! 

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