Discovering Precision Medicines to Treat Kids’ Rare Diseases

Fact: Sick children need safe & effective medicines (i.e., precision medicine) to fight their disease, get well, enjoy a good life, and go out to play! We were all children once, and we survived childhood!

Unfortunately, for children diagnosed with rare diseases, chances are that there may be no medicine as treatment! The status quo in pediatric rare disease medicine is unacceptable: for 95% of the known 7000 odd rare diseases that afflict children, there are no exact medicines. The numbers add up: Nearly 20 million kids in the USA and about 200 million kids globally are afflicted with rare diseases; 30% of kids afflicted with rare diseases will not live to see their 5th birthday.  All this leads to immense suffering for sick kids, their families and caring communities. Watch this video: AMC #Meds4Kids which tells the story of two children and their families afflicted with rare diseases.

An important reason for this this glaring gap in children’s medicine may be attributed to the lack of profitability for pharmaceutical companies. No market-based drivers means that essential research and development needed to create precision medicines for kids don’t attract the investment dollars; more lucrative R&D projects win the financial backing.

Listen to what Gabriella Miller said about medicines needed for children afflicted with rare diseases.

When we think outside the box, we know that there has got to be a way to help sick kids; profitability should not stand in the way of healing sick children. Children’s lives are as valuable as adult lives. This is why AMC embraces a NONPROFIT mode to carry out collaborative R&D efforts to create and provide precision medicines for kids! We can do it!

How? As a nonprofit corporation, AMC is not tossed about by market forces, and there are no distractions of profitability; we simply focus on the kids who desperately need the medicines. Whether a disease has 20 or 200 or 1000 kids diagnosed globally, AMC can SERVE them by bringing forward essential medicines to treat/cure their disease. The possibility to heal sick kids is AMC’s motive and inspiration.

We must help these children because we can: the necessary modern technology is available; skills and talent are plentiful; the need is huge. Money is necessary to create these important medicines.  And that is why we come to you for the essential financial support so AMC can carry out collaborative research and bring forward precision medicines for sick kids. We believe sick children with rare and neglected diseases must also benefit from powerful modern drug discovery technology. We invite you to participate in this mission, and with your financial support for AMC, we can together help sick kids. It is necessary that we lean in a little closer to these sick children to hear their silent and desperate cries. AMC hears the cries of sick kids, and knows that their lives are valuable. You have the power to make a huge difference in the lives of these sick kids–where there is no medicine now, you help create healing medicines by supporting AMC #Meds4Kids

Here is some good news: The same technology used today in pharmaceutical industry to find medicines for adult common diseases (e.g., high cholesterol, cardiovascular, respiratory, etc.) can also be used to find medicines for childhood rare diseases. That is why we at American MedChem Nonprofit Corporation  (AMC) feel optimistic about unleashing the power of modern drug discovery technology and the talent of research scientists to create novel, safe and effective medicines (Precision Medicines) specifically for children with rare/neglected diseases.

Source: icnw