Home2020/04 April 29, 2020 -

https://mysicklefamilyllc.com/wp-content/uploads/2020/04/shutterstock_381735667.jpg

FFF Enterprises and BioNews announced today that both rare and orphan disease advocates are joining forces to provide patients with resources to help them connect as a community and continue to manage their health during this time when many are finding themselves alone. Recognized as the nation’s leading supplier of critical-care biopharmaceuticals, plasma products and vaccines, FFF has been pro-active in communicating with pharmaceutical manufacturers across the country, ensuring that patients do not experience interruptions...

https://mysicklefamilyllc.com/wp-content/uploads/2020/04/shutterstock_220041373-1400x480@2x-2-1280x720.jpg

“What’s wrong with you now?” I am all too familiar with that phrase. From a young age, I have often felt like I’ve had to prove myself in regards to my health. As if this disability isn’t enough, I’ve also had to deal with others’ opinions. Sickle cell can be very unpredictable. A sickle cell crisis can be triggered by a variety of factors, including temperature, stress, and dehydration — not to mention mystery triggers....

https://mysicklefamilyllc.com/wp-content/uploads/2020/04/shutterstock_418713622_zpsry953y6x.jpg

Fera Pharmaceuticals submitted an application to the U.S. Food and Drug Administration (FDA) requesting orphan drug status for naproxcinod, a potential treatment for for sickle cell disease (SCD). Orphan drug designation is given to experimental treatments for rare diseases (those that affect fewer than 200,000 people in the U.S.) to support their development. The designation also comes with certain benefits, including financial incentives for clinical development and commercialization, special fee exemptions, as well as seven-year...

https://mysicklefamilyllc.com/wp-content/uploads/2020/04/JennPowell-telemed-579x480@2x.jpg

While there are few silver linings to the cloud created by COVID-19, the pandemic that has killed tens of thousands, hobbled economies worldwide and drove millions to quarantine in their homes, one may be a new appreciation of telemedicine. “If something good could come out of this crisis, it’s that we would learn how valuable telehealth could be to our community,” said Steven Shook, a neurologist at the Cleveland Clinic in Ohio. Shook specializes in neuromuscular disorders...

https://mysicklefamilyllc.com/wp-content/uploads/2020/03/shutterstock_385795960_zpswoacxqyp-1024x720.jpg

The National Organization for Rare Disorders (NORD) has opened a financial assistance program for people in rare disease community who are affected by the COVID-19 pandemic in the U.S. Called the NORD COVID-19 Critical Relief Program, the effort will provide up to $1,000 annually to those eligible to support critical, non-medical needs. The program is designed to help rare disease patients and their families who may be facing monetary hardships due to the outbreak. “Providing financial...