Home2020April 2020 -

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APCER Life Sciences has released a report confirming the safety profile of Endari (L-glutamine), according to Emmaus Life Sciences, which developed the therapy. Endari is an oral therapy for sickle cell disease (SCD) approved by the U.S. Food and Drug Administration to reduce severe complications associated with the disease in both adults and children, ages 5 and older. The medication works by increasing the amount of free L-glutamine. L-glutamine is an amino acid — the building...

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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...

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“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....

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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...

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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...

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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...

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A team at the Case Western Reserve University has been awarded up to $3.7 million from the National Heart, Lung, and Blood Institute (NHLBI) to conduct early studies of emerging gene therapies for sickle cell disease (SCD). Led by Umut Gurkan, PhD, the team will examine blood samples collected from SCD patients before and after they receive gene therapies, and test them for improvements in red blood cells. These potential gene therapies work by modifying a...

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In the early days of this outbreak, only a couple of media outlets nonchalantly reported the events surrounding COVID-19. Back then, we had no idea how serious this virus was. The word coronavirus wasn’t even an active part of my vocabulary. Fast-forward a few months later and it is all people talk about. It’d be dishonest to say this coronavirus outbreak doesn’t worry me. “Worried” is even too light of a word. I have been...

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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...

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“Survival of the fittest” is a phrase I have come to loathe. This manner of thinking is particularly appalling to hear as a sickle cell patient. I have heard the phrase more frequently lately, against the backdrop of the current COVID-19 pandemic. But what does it mean? Historically, the phrase was derived from Darwinian evolutionary theory and speaks to the process of natural selection. Natural selection states that characteristics advantageous to the survival of a...