By 2029, Americans with sickle cell disease (SCD) could be living 10 years longer than they do today, predicts U.S. Health and Human Services Secretary Alex Azar.

That’s one of the Trump administration’s long-term goals, Azar — the nation’s top health official — told more than 900 delegates attending the 2019 NORD Rare Diseases & Orphan Products Breakthrough Summit in Washington, D.C.

The recent summit, organized by the National Organization for Rare Disorders, also featured speeches by Norman E. “Ned” Sharpless, MD, acting commissioner of the U.S. Food and Drug Administration (FDA), and his predecessor, former FDA Commissioner Scott Gottlieb, MD. In his 17-minute speech, Azar used SCD as an example of President Trump’s commitment to those with rare diseases.

The National Institutes of Health plans to invest at least $100 million over the next four years toward an audacious goal: develop affordable, gene-based cures for sickle cell disease (SCD) and HIV. The Bill & Melinda Gates Foundation will also invest $100 million toward this goal. The intention is for these cures to be made globally available, including in low-resource settings.

This initiative follows a bold announcement made earlier this year by President Donald J. Trump during the State of the Union Address to end the HIV epidemic in the United States in the next 10 years. Ending the HIV Epidemic: A Plan for America(link is external) aims to leverage the powerful data and tools now available to reduce new HIV diagnoses in the United States by 75% in five years and by 90% by 2030. The Trump Administration has also elevated the attention paid to sickle cell disease, identifying it as an intractable health challenge with the potential for dramatic advances in the coming years.

The Centers for Disease Control and Prevention (CDC) will share an award total of nearly $1.2 million with institutions in seven U.S. states, allowing them to take part in a data gathering program on sickle cell disease and how it affects daily life.

The funding brings to nine the number of states in the CDC’s Sickle Cell Data Collection program. The new states join Georgia and California as CDC partners in efforts to gain population-based, comprehensive health information. The program is aimed at better understanding the needs of sickle cell disease (SCD) patients, and in developing therapies more suited to their needs and providing greater support.

“HHS is committed to normalizing the lives of people living with sickle cell within 10 years,” Brett Giroir, MD, assistant secretary for health at the Department of Health and Human Services, which includes the CDC, said in a news release.

The regulatory approval of a groundbreaking new Sickle cell disease drug in the United States by the Food and Drugs Administration (FDA) sparked off plenty of hope and optimism earlier this month and the news that it is already heading for clinical trials in some African countries was also welcomed. But that optimism has been replaced by concerns the new treatment is priced beyond the reach of the majority of sufferers on the continent.

Swiss pharma giant Novartis announced this month the US FDA approved its new drug crizanlizumab, marketed as Adakveo, for adults and patients over 16 to start using the medication in a matter of weeks. In recent years Hydroxyurea has been the standard-of-care drug for the treatment of sickle cell disease’s pain crisis.

James Franklin is Pennsylvania’s highest-paid state employee. He agreed to a six-year contract extension last winter that will pay him $35.4 million through 2025. He doesn’t expect any sympathy because of a personal sacrifice he will make to coach Penn State this season, if there is a college football season. He doesn’t need sympathy. He doesn’t want it.

But that doesn’t change the fact that this fall will be especially difficult for Franklin.

“A lot of heartache over it,” Franklin told HBO’s “Real Sports” last week.

The younger of Franklin’s two daughters, Addison, has Sickle cell disease. To protect her from being exposed to the coronavirus, Franklin and his wife, Fumi, have made a tough decision. He will spend the season with his team. His wife will stay at the family home in Destin, Fla., with Addison, who turned 12 in March, and their other daughter, Shola. 

Sickle cell disease (SCD) is an inherited group of blood disorder which is genetic in nature. It is usually transferred from parents to the child during birth i.e. both the parents can be carriers of SCD. Healthy RBCs are round in shape, which moves through small blood vessels and carries oxygen to all parts of the body. In someone who has SCD, the RBC becomes sticky and hard and starts to look like C- shaped similar to that of a farm tool ‘sickle’.

The sickle cells die early, which causes a constant shortage of RBC, which leads to low oxygen carriers in the body, mentioned Dr Rahul Bhargava, director and head, haematology and bone marrow transplant, Fortis Memorial Research Institute, Gurugram.

How can Implementation science frameworks help implement newborn screening in African settings by Lewis Hsu, MD, PhD. Lewis Hsu is a professor of pediatrics and director of the pediatric sickle cell at the University of Illinois at Chicago.

Learning Objectives:
• Recognize key features of newborn screening for SCD in several countries
• Discuss barriers and resources for large-scale newborn screening for SCD in your local region
• Categorize barriers and resources to large-scale newborn screening for SCD for different stakeholders using Getting To Outcomes framework
• Predict how stakeholders interests and resources could be aligned to promote successful large-scale newborn screening for SCD

Date and Time:
Mon, 17 Aug 2020, 17:30 IST

Location:
Online Event

This event has ended.

Join us for our next 1-hour virtual educational webinar led by Versiti experts that will provide facts about our blood mission and enables community members & advocates to ask questions while sharing their perspectives on building community bonds through blood donorship.

Versiti Speakers:
• Manager of Donor Services, Denena Edwards
• Manager of Diversity Partnerships, Mary Jones
• Director of Clinical Education, Sue Johnson
• Field Marketing Manager, Transfusion Medicine, Greg Asmus

Date And Time:
Wed, August 19, 2020
3:30 AM – 4:30 AM IST

This event has ended.

Kids Conquering Sickle Cell Disease Foundation is excited to offer a new virtual transition education program with support sessions for adolescents, their parents, and young adults starting in August to December 2021. Our goal is to increase the number of emerging adults who successfully transition into adult care and provide support to prepare for adult life with a chronic condition.

The interactive session topics will include, but not be limited to, balancing life with sickle cell or related blood disease, healthy habits, warning signs of complications, post high school planning (college/workforce, nutrition, social life), mental health, managing pain, sexual health, organizing health care information, planning for emergencies away from home, etc.

Date and Time:

Session 1:
Saturday, July 10, 2021
10:00 AM - 12:00 PM EDT

Session 2:
Tuesday, August 10, 2021
10:00 AM - 12:00 PM EDT

Session 3:
Friday, September 10, 2021
10:00 AM - 12:00 PM EDT

Session 4:
Sunday, October 10, 2021
10:00 AM - 12:00 PM EDT

Session 5:
Wednesday, November 10, 2021
10:00 AM - 12:00 PM EST

Session 6:
Friday, December 10, 2021
10:00 AM - 12:00 PM EST

Location:
Online Event

The COVID-19 pandemic threatens to cause a blood supply shortage in the United States, and as a result, clinicians may be compelled to ration or conserve blood products. For people living with sickle cell disease (SCD), blood transfusions are often necessary to decrease the frequency of severe symptoms.

In a new Blood paper, Michael R. DeBaun, MD, MPH, of Vanderbilt University Medical Center, Vanderbilt-Meharry Sickle Cell Disease Center of Excellence, suggests that while monthly blood transfusions are the standard method of stroke prevention for children with SCD and at high risk of strokes, initiation of hydroxyurea therapy may be an effective alternative, if the monthly blood transfusions are interrupted.