Help Find the Cure for Niemann-Pick Disease #NPD #NiemannPickDisease #cure - http://clapway.com/2015/08/25/help-find-the-cure-for-niemann-pick-disease-234/

Luckily for future holders of Niemann-Pick Disease Type C (or NPC) in their hereditary makeup, the National Niemann-Pick Disease Foundation (NNPDF) is poised to kick this fatal genetic mutant down in its own path. And, the NNPDF has a lot of help…

What is Niemann-Pick Disease?

Niemann-Pick Disease, caused by genetic mutations, is one of many lysosomal storage diseases affecting metabolism. The most common types — Niemann-Pick Types A and B (ASMD or Acid Sphingomyelinase Deficiency) and Niemann-Pick Disease Type C (NPC) — affect the population indiscriminately. A higher incidence happens to occur in the following populations: the Ashkenazi Jewish population, French Canadian population of Nova Scotia, Maghreb region (Tunisia, Morocco, and Algeria) of North Africa, and the Spanish-American population of southern New Mexico and Colorado.

Niemann-Pick Disease Type C (NPC)

The National Niemann-Pick Disease Foundation (NNPDF) is on a quest to find a cure for all strains of Niemann-Pick Disease. However, its primary target has been Niemann-Pick Type C or NPC for many reasons.

Niemann-Pick Type C (NPC) is different from Type A or B (ASMD). Patients with NPC are unable to metabolize any cholesterol or other lipids properly. As a result, excessive amounts of cholesterol accumulate within the patient’s liver, spleen, and even the brain.

Furthermore, NPC is always fatal. Children with Niemann-Pick Type C usually die before 20 year of age (many do not make it to age 10). When the onset of symptoms comes later in life, it is extremely rare for anyone with NPC to reach age 40. Oftentimes, the disease can also be misdiagnosed as a learning disability, due to the symptoms it causes, such as mild retardation, “clumsiness,” and the delayed development of fine motor skills. As such, it is common for diagnosis to escape families and physicians for many years.

The Quest to Find a Cure for NPD

The NNPDF supports and promotes research with the goal of finding treatments and a cure. It also remains a hub for support for individuals and families affected by NPD. Ultimately, its vision is to create a world where NPD can no longer threaten the prospect of a full and productive life for patients and their loved ones.

As reported by The Wall Street Journal, not only are the NNPDF’s activist efforts appreciated, but they’re heavily supported by not one, but three companies, ready for a round of clinical trials meant to develop treatment and therapy for NPC. This all sounds good and dandy, but one issue remains: Can this medical research community support more than one trial at a time, even with such welcomed support?

Niemann-Pick Type C Could Be Too Rare 

As luck would have it, Niemann-Pick Type C or NPC is a rare disease. Its rareness makes it difficult to find an appropriate number of afflicted patients in order to test and demonstrate results. In fact, the number of patients with Niemann-Pick Type C is approximately 500 all around the world. Since the pool of patients with NPC is (thankfully?) small, pharmacists and doctors say the results may not be reliable.

“This is a critical juncture for our community,” said Nadine Hill, executive director of the National Niemann-Pick Disease Foundation. “To lose out on having an approved treatment is our worst-case scenario.”

This is the outcome the community is trying to avoid. Scientists, doctors, and family are worried that there will not be a trial with the numbers to get a drug approved by the Food and Drug Administration (FDA) for either treatment nor a possible cure. The FDA shared that 17 of the 41 new drugs it approved in 2014 for rare diseases.

Even if the desired number of patients might be reached, some are reluctant to participate in the trials as they can require heavy travel and time off from work or school. This makes activism, funding, support by and for the National Niemann-Pick Disease Foundation (NNPDF) crucial.

Show your support here. Your attention has the potential to save lives.

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Help Find the Cure for Niemann-Pick Disease

Drug Typically Used for Arthritis Possibly Treats Vitiligo Too - http://clapway.com/2015/06/26/drug-typically-used-for-arthritis-possibly-treats-vitiligo-too323/

A recent experiment which tested a middle-aged woman suffering from progressive vitiligo, was shown to have resulted in most of her patched skin turning back to its original color. This was all thanks to an oral drug, tofacitinib, usually meant for treating severe cases of rheumatoid arthritis.

About the drug in question for treating vitiligo

The drug in question, tofacitinib, “belongs to a family of drugs known as Janus kinase (JAK) inhibitors that have been approved… by the US Food and Drug Association (FDA)” according to recent reports. This drug is typically prescribed to those who are diagnosed with moderate to severe rheumatoid arthritis. Additionally, when it comes to degenerative diseases such as vitiligo, tofacitinib has also been proven to treat alopecia, a condition which results in hair loss.

The experiment conducted for testing vitiligo

One 53-year-old woman was tested to see if her progressive vitiligo can be treated with the use of the arthritis drug, tofacitinib. The experiment was conducted by having her take 5mg of the oral drug once a day, the dosageeventually being increased by 5mg about a month later. Taking a dose of approximately 10mg of tofacitinib proved show success after two months as the woman’s white patches were showing partial repigmentation. According to researchers of the study, “After 5 months, the white patches she had been most concerned with on her face and hands were nearly all gone. Only a few white spots remained on other parts of her body”. It appears with this experiment/trial, that this discovery is definitely, and truly a breakthrough, when it comes to an autoimmune disease, such as vitiligo, finally having a potential medication to help treat the horrendous depigmentation.

Any long-term and adverse effects at the moment?

During, and after, the experiment, there appeared to be no long-term, adverse effects when the woman was on the tofacitinib trial, for her vitiligo. However, because only one woman was used during the trail there would need to be a lot more people recruited for further findings. This is to make sure the findings are more concrete and is safe, as well as truly an appropriate treatment for vitiligo, for sufferers of the autoimmune disease to benefit from.

For more information on this experiment/trial, please go to JAMA Network.

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Drug Typically Used for Arthritis Possibly Treats Vitiligo Too

Beat the Hangover Like a True Mexican - http://clapway.com/2015/06/09/beat-the-hangover-like-a-true-mexican/

Have you ever had a night out, party and then when you wake up in the morning with the worst headache and thirst, you say to yourself never again? As you probably know Mexicans love to party, and oh boy we do it well. Some well-known ammo or staples include tequila, margaritas, mezcal and certainly mariachis.

But as all good things, there is always a cost, and after a night out with more than a fair share of tequila shots, you might end up having a miserable “cruda” or hangover. Good thing that there are certain Mexican recipes that can get you back on track, people know them by heart and rely on them t after a night out. While in most cities, restaurants and bars close sometime after 2 AM, in Mexico City you can drive and find many eating places where the peak hours start precisely after this time. These are some of the most popular dishes you might find in these restaurants:

Chilaquiles

The most common and popular dish for breakfast after a long evening. Chilaquiles are made up of fried tortilla pieces (please don’t think of using Doritos), green or red salsa, cream and cheese. It can also be accompanied with some black beans or even a fried egg on top if you are truly hungry. Urban myth says that the spicier the chilaquiles the more they cure the hangover because the spicy food makes you thirsty, which combats the dehydration since you tend to drink more. If you ever go to a Mexican wedding don’t be surprised when chilaquiles are served around 6 am to prevent a bad hangover the next day.

Micheladas

This drink is prepared with beer, salt, lime and tomato juice. Its important to add tomato juice since this has magnesium, potassium and some antioxidants that help eliminate toxins from your body. Micheladas are just the hangover cure for you.

Pozole

Pozole from the nahuatl pozolli, is a classic in the western states of Guerrero and Michoacán in Mexico. This stew with corn and (beef or pork) meat has several variants depending on each state. You can find green, red and white pozole and still remains a popular hangover cure.

Menudo

A labor intensive soup made out of beef tripe and a spicy broth. Menudo can have some variants depending on what part of Mexico you are located. In northern Mexico people add unground tortilla corn and in the northwest the dish is made without the red chiles and called menudo blanco.

What other home remedies to cure hangovers have you experienced? Don’t be shy and share them with us! For more information and tips on traveling and food in Mexico and abroad visit michelleonbell.com.

Need a hangover cure after drinking on a week night? Try SensorWake for the best of wake up calls on the worst kinds of mornings:

Beat the Hangover Like a True Mexican