Medunik USA, a company dedicated to improving the health and quality of life of Americans with rare diseases by making orphan drugs available in the U.S., announced today that Siklos® (hydroxyurea), an FDA-approved orphan drug, is now available in 100 mg tablets for pharmacies to order.

Siklos® is indicated to reduce the frequency of painful crises and the need for blood transfusions in pediatric patients, two years of age and older, with sickle cell anemia with recurrent moderate to severe painful crises. It is the first and only FDA-approved hydroxyurea-based treatment for use in pediatric patients with sickle cell anemia.

Medunik USA holds the exclusive rights to market and distribute Siklos® in the United States, through an agreement with ADDMEDICA, a French pharmaceutical company specializing in hematology.

A significant proportion of children with sickle cell disease (SCD) are not receiving key recommended vaccines on time or at all, according to a new study examining pneumococcal and meningococcal vaccinations among children in Michigan.

The study, “Pneumococcal and Meningococcal Vaccination among Michigan Children with Sickle Cell Disease,” was published in the Journal of Pediatrics.

The researchers sought to determine the proportion of children with SCD compared to kids without the condition in Michigan who were vaccinated according to pneumococcal vaccination recommendations and, secondarily, to examine meningococcal vaccination.

The study included children born in the state of Michigan between April 1, 1995, and January 1, 2014. A total of 1,022 children with sickle cell disease were matched to 3,725 children without SCD using age, sex, race, and zip code.

Physicians who use stigmatizing language in their patients’ medical records could be affecting the care they receive for years to come, according to a new Johns Hopkins study.

Titled “Do Words Matter? Stigmatizing Language and the Transmission of Bias in the Medical Record,” and published in the Journal of General Internal Medicine, the study aimed to determine whether the language and descriptions used in patient records could perpetuate bias among doctors.

The study analyzed the notes and descriptions physicians had written during visits to a hypothetical patient at a large urban medical center. Researchers then assessed how the language used in the notes influenced the patient’s care and pain management.

Researchers at the Johns Hopkins School of Medicine used an experimental vignette study design, in which 413 residents and medical students were randomized to read one of two chart notes presenting medically identical information about a hypothetical 28-year-old patient with sickle cell disease (SCD) and chronic pain in his left hip.

Music therapy can help ease pain in adults with sickle cell disease (SCD), a pilot study showed. A single session of music improvisation with a music therapist helped significantly diminish the intensity of patients’ pain and uplift their moods, the study noted.

The research, titled “The Effects of a Single Electronic Music Improvisation Session on the Pain of Adults with Sickle Cell Disease: A Mixed Methods Pilot Study,” was published in the Journal of Music Therapy.

Many patients with sickle cell disease, particularly adults, often experience episodes of pain, including acute pain crises, chronic pain, and neuropathic (nerve) pain.

Pain can result from chronic inflammation due to the obstruction of blood circulation by sickled red blood cells. Organ damage and increased sensitivity to pain due to the use of opioids as painkillers also can occur.

Other contributors include psychological, social, cultural, and spiritual factors.

The Sickle Cell Foundation of Georgia (SCFG) has partnered with Biotech Research Laboratories and the Phoenix Tears Foundation to raise money for the first clinical study focused on the effectiveness of cannabis oil in sickle cell disease (SCD)-related pain management.

The initiative is in honor of World Sickle Cell Day, held annually on June 19 to mark the day the United Nations General Assembly recognized SCD as a public health problem.

“We urge the Sickle Cell community, our friends and allies to join in supporting the coalition as we search for alternative treatments for those battling this painful and sometimes fatal condition,” Deb McGhee McCrary, SCFG president and CEO, said in a news release. “These trials will fully explore the pain relief properties of medical cannabis as an alternative to highly addictive opioids.”

Emmaus Life Sciences, Inc., announces it has entered into a distribution agreement with McKesson Plasma and Biologics LLC (McKesson), a division of McKesson Corporation. The agreement makes Endari™ (L-glutamine oral powder), indicated to reduce the acute complications of sickle cell disease in adult and pediatric patients 5 years of age and older, available nationwide to pharmacies that utilize McKesson as their primary and exclusive distributor. McKesson is the largest healthcare distribution company in the world.

Since launching Endari, the first U.S. FDA approved sickle cell treatment in 20 years, Emmaus has brought this therapy to patients in over 30 states, Washington DC and Puerto Rico. In addition to the distributors already in place, this agreement with McKesson will further broaden Endari’s distribution, with the potential to reach many more patients suffering from the acute complications associated with sickle cell disease.

“As the demand for Endari continues to increase, we seek to build our distribution network to ensure that we reach patients in need,” said Yutaka Niihara, MD, MPH, CEO and Chairman of Emmaus.

September is National Sickle Cell Awareness Month, designated by Congress to help focus attention on the need for research and treatment of sickle cell disease.

SCDAA’s theme for this year is Sickle Cell Matters 2021. You can share our Awareness Month Flyer & Facts Sheet as part of your awareness efforts or join Sickle Cell Disease Association of America (SCDAA) at one of the many great events to support sickle cell awareness! Our hashtags this year will be #SickleCellMatters2021, #SickleCellAwarenessMonth, #SCDAA2021AwarenessMonth and #SCDSCTmatters.

We encourage everyone to be a part of this national effort to increase awareness about sickle cell disease and sickle cell trait during the month of September. Individuals and organizations can join our efforts to bring attention to sickle cell disease by engaging elected officials for proclamations, hosting awareness events, distributing educational information to dispel the myths about sickle cell disease, and lighting public spaces, buildings and landmarks red!

George H. Carter appears to have won his fight to ensure that people like himself who suffer from sickle cell disease can get the high dosages of opioids needed to control the excruciating pain.

On June 15, Gov. Ralph S. Northam approved a regulation to make a change that Mr. Carter pushed as administrator and chief lobbyist for the nonprofit Sickle Cell – Virginia, the umbrella state-wide organization with chapters in Richmond and other localities.

The regulation change will allow physicians to provide those with the niche genetic blood disease with far higher levels of opioids without being forced to justify the treatment as is now required when such potentially addictive drugs are prescribed to those in pain.

When 9-year-old Jeremy Brown is in pain, it feels like he is being stabbed, while the pain experienced by Deborah Oliver, 40, is like a hundred simultaneous charley horses.

Brown, of Bridgeport, and Oliver, of New Haven, have sickle cell disease (SCD), a genetic blood disorder that causes excruciating pain, life-threatening complications and a shortened life expectancy. Almost one-half of sickle cell patients die in their 40s.

The disease affects some 100,000 Americans, about one in 365 African Americans and one out of 16,300 Hispanics; and in lesser numbers, people with Middle Eastern, Indian, Caribbean and Mediterranean ancestries. An estimated 2,000 people in Connecticut have SCD.

But the disease—discovered over 100 years ago—receives little research, funding and attention.

Twin boys from the Richmond area are taking on your lawmakers to make sure kids who are in the hospital get the support they need from Washington. 

Gabriel and Noah Cypress look like your average 9-year-olds. But when they were born, mom LaToya Cypress says the doctors told them something shocking - the twins had sickle cell disease.

This year, Gabriel and Noah were invited to go to Washington, D.C. to talk to representatives about child healthcare during the Children Hospital Association’s Family Advocacy Day.

The boys also spoke with Sen. Mark Warner (VA-D), Representatives David Brat (District 7-R) and Donald McEachin (District 4-D), telling them what it’s like to be in the hospital so often. They also thanked the lawmakers for reauthorizing CHIP funding this year.