What are the most well - known orphan drug success stories in recent years?

Falguni Nayar. She founded Nykaa, an Indian e - commerce beauty retailer. She saw the potential in the beauty market in India and built a successful business. Nykaa has become a household name in India, providing a wide range of beauty products and also promoting Indian beauty brands.

In the field of spinal surgery, there was a patient with a severely herniated disc that was causing intense pain and limited mobility. After a minimally invasive spinal surgery, the patient's pain disappeared almost immediately. Within a few weeks, they could move freely and resume their daily activities like walking long distances and doing light exercise.

A recent well - known RPG horror story involves a new RPG where players found that their in - game avatars were being controlled by an unknown force for a brief moment. It was really scary as they could see their characters move on their own and perform actions they didn't initiate.

In the field of cancer, drugs targeting the epidermal growth factor receptor (EGFR) are a success. By analyzing the structure of EGFR, drugs such as erlotinib were developed. These drugs bind to the receptor and inhibit its tyrosine kinase activity, which is often overactive in certain types of cancer. This helps in slowing down the growth and spread of cancer cells. It was the understanding of the receptor's structure that enabled the design of such effective drugs.

One recent success story is that of a financial analyst who used candlestick patterns to analyze stocks. He identified a 'hammer' pattern in a stock that had been in a downtrend. This pattern often signals a potential reversal. He bought the stock based on this pattern and within a few weeks, the stock started to rise and he made a profit. Candlestick patterns can be very effective when used correctly in technical analysis.

Well, there's the off - label use of immunosuppressant drugs like tacrolimus in treating atopic dermatitis. Tacrolimus was developed mainly for preventing organ transplant rejection. However, when applied topically, it has been effective in reducing the inflammation and itchiness associated with atopic dermatitis. Additionally, the off - label use of statins for non - lipid - related conditions such as reducing inflammation in rheumatoid arthritis is also an interesting development. While statins are mainly for cholesterol control, emerging research suggests they may have a role in modulating the immune response in arthritis patients.

The discovery of new drugs for Hepatitis C is also a big deal. Scientists were able to develop drugs that directly target the virus and can cure Hepatitis C in a matter of weeks in most cases. This was a huge step forward compared to the previous treatments which had many side effects and were less effective.

In the casino world, the martingale system has had some short - term success stories. For example, there have been tales of players using it on simple even - money bets in games like baccarat. They double their bets each time they lose until they win. Some players have managed to walk away with a small profit in a single session.

Well, the Theranos case is also notable. Elizabeth Holmes claimed that her company had developed a revolutionary blood - testing technology that could perform multiple tests with just a small amount of blood. However, it turned out that the technology didn't work as promised. She managed to raise a large amount of capital based on false claims, fooling many investors and high - profile individuals for a significant period of time.

Sure. A notable one is pembrolizumab. It was developed for cancer treatment and has been very successful in treating various types of cancers like melanoma, lung cancer etc. Another recent success is the repositioning of a drug for treating rare genetic diseases. For example, certain enzyme - replacement therapies that were initially developed for one rare disease are being explored for other related rare diseases as well.