SCDAA's First Masterclass on the Cusp of a Cure
All Things Considered – SCD Treatment: A Personal Choice
Thursday, August 24, 2023 at 3-4 p.m. EST
Co-hosts Teonna Woolford and Dr. Wanda Whitten-Shurney will discuss the many considerations that go into choosing disease modifying treatments or curative therapies. Panelists include speakers who will share their lived SCD experiences, the choices they made, and why.
BOSTON & ZUG, Switzerland--(BUSINESS WIRE)--Jun. 8, 2023-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) and CRISPR Therapeutics (Nasdaq: CRSP) today announced that the U.S. Food and Drug Administration (FDA) has accepted the Biologics License Applications (BLAs) for the investigational treatment exagamglogene autotemcel (exa-cel) for severe sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT). The FDA has granted Priority Review for SCD and Standard Review for TDT and assigned Prescription Drug User Fee Act (PDUFA) target action dates of December 8, 2023, and March 30, 2024, respectively. Updated data from the pivotal trials supporting the regulatory submissions will be presented at the Annual European Hematology Association Congress on June 11, 2023.
[Hanover, Md., June 26, 2023] – The Sickle Cell Disease Association of America, the leading patient organization for people with sickle cell disease, announced a pilot program with MedicAlert Foundation to enhance the safety and well-being of people living with sickle cell disease.
More than 100,000 Americans live with sickle cell disease, a genetic blood disorder that affects red blood cells. Acute pain episodes known as sickle cell crises are one of the most common and debilitating symptoms of sickle cell disease. These crises can be unpredictable and extremely painful, lasting from a few hours to a few weeks. They’re the No. 1 reason people with sickle cell disease seek emergency treatment.
However, patients seeking treatment for a sickle cell crisis face hurdles to getting the care they need in the emergency department. Many emergency physicians are not well versed in sickle cell disease and treatment protocols. And with the rise of the opioid crisis, emergency department providers are cautious about providing the powerful pain medication needed to quell a sickle cell crisis. Sometimes sickle cell patients are unfairly labeled as “drug seekers” by emergency department personnel who don’t understand the disease.
The Sickle Cell Disease Association of America and MedicAlert pilot program aim to improve access to timely, effective emergency care for people experiencing a sickle cell crisis. MedicAlert Foundation is a leading nonprofit providing lifesaving medical identification and emergency response services for millions of people living with chronic health conditions.
One of the most significant events of 10th Annual Warriors Convention was the 2021 to 2023 Graduation Ceremony, where we celebrated the graduating class of the last three years. It was a joyous occasion to see warriors and caregivers from Pre-K to post-graduate celebrating such an important milestone virtually and in person.
The graduation ceremony was a testament to the graduates' resilience, strength, and determination. As a community, we take pride in supporting and uplifting each other in their journey toward success. We are creating a 2021 to 2023 Graduating Class Yearbook for this special occasion. We invite all graduates to be featured in this yearbook by filling out the form linked below before August 25th, 2023.
We also invite you to share your encouraging words with our graduates. Your kind words will undoubtedly inspire and motivate them to reach greater heights. Please fill out the form linked below if you'd like to include a message to your graduate or the graduating class.
We are thrilled to celebrate the accomplishments of the 2021-2023 graduates of the sickle cell community and look forward to hearing from you. If you have any questions, don't hesitate to contact Dominique Goodson at dgoodson@sicklecellconsortium.org.
Date & Time
Sep 28, 2023 11:00 AM in Eastern Time (US and Canada)
Description
The theme for this year's summit is "Thriving with Sickle Cell Disease: Optimizing Care Through all Stages of Life."
The goal is to highlight the latest trends, successes, challenges, and barriers to propel the lives of the Sickle Cell community beyond survival/sustenance to thriving.
Keynote speaker: Wanda Whitten-Shurney, CEO and Medical Director of SCDAA-MI
Session 1: Genetics and Foundations of Care Featured speakers - Kay Saving, Marwah Farooqui, Lewis Hsu
Session 2: Emerging Care, Medicines, and Treatments Featured speakers - Franklin Njoku, Damiano Rondelli, Joseph Colla
Session 3: Building Community Partnerships through Training
We are offering a maximum of 4 CE credits for providers. Please indicate your request for credits in the exit survey.
The summit is free for all participants.
We enthusiastically invite you to The Sickle Cell Association of New Jersey's 11th Annual Statewide Sickle Cell Symposium on Saturday, November 18th from 9:00am to 2:30pm at the Liberty Science Center in Jersey City.
Our goal is to improve and expand patient and provider knowledge and to improve the treatment for sickle cell disease. We are excited to host a Pre-symposium Student Research Fair at 9:00am featuring poster presentations on Comparative Effectiveness Research.
We will also host the Lunch and Learn, the Youth Research Expedition and museum admission to the exhibit halls.
We're planning an exciting program suitable for individuals effected by sickle cell disease, health care professionals who provide care for patients with the condition from infancy to adulthood (including private practice physicians, hospitalists, ED staff, nurses, social workers), and the community at large.
Early Bird Registration until November 17, 2023 is FREE. Late Registration--Day of Event is $25.00. Parking validated. In addition, be sure to Get Connected with the SCANJ. Patients who are registered with us receive priority for special programs and resources. For more information contact us at 973 482-9070.
The Dr. Charles F. Whitten Awards for outstanding service will be announced at the Symposium.
Hematopoietic stem cell transplant for sickle cell disease from HLA-matched sibling donor (MSD) after myeloablative conditioning has been proven as curative therapy for sickle cell disease for over 25 years. It is no longer considered an experimental procedure. Over 2,000 transplants have been performed successfully across the world from HLA-matched sibling donors. Pooled Overall Survival rates for children and adults are high at 97% and 98%, respectively (meta-analysis by Iqbal 2021). The outcomes in sickle cell disease are much better for younger ages, but outcomes can still be good for selected adults. MSD HCT is expensive, but multiple analyses have shown that long-term health care costs go down, quality of life improves and organ damage is usually slowed or stopped. This means that MSD HCT is very cost-effective for the family, the health care system and society in general (less than $50k per QALY). Therefore, it is logical for MSD HCT to be covered by health insurance.
The worldwide pandemic of COVID-19 (SARS-CoV2) infections seem to have settled down, and we are now seeing waves of infections with new variants. As facemasks come off and isolation rules end, everybody has higher chances of catching other respiratory infections, such as RSV (respiratory syncytial virus) and influenza (flu). SCDAA’s Medical and Research Advisory Committee (MARAC) endorses a shared-decision-making process about vaccines and treatments but would like to offer unbiased information to help in the decision-making process.
Rare Disease Day® takes place worldwide, typically on or near the last day of February each year, to raise awareness among policymakers and the public about rare diseases and their impact on patients’ lives. Since 2011, NCATS and the NIH Clinical Center have sponsored Rare Disease Day at NIH as part of this global observance. Rare Disease Day at NIH aims to raise awareness about rare diseases, the people they affect, and NIH collaborations that address scientific challenges and advance research for new treatments.
The Goals of Rare Disease Day at NIH Are to:
• Demonstrate the NIH commitment to helping people with rare diseases through research.
• Highlight NIH-supported rare diseases research and the development of diagnostics and treatments.
• Initiate a mutually beneficial dialogue among the rare diseases community.
• Exchange the latest rare diseases information with stakeholders to advance research and therapeutic efforts.
Date & Time:
Tuesday, February 28, 2023
9:00 AM to 5:00 PM EST
Congressional Briefing:
Comprehensive Sickle Cell Disease Care in a Time of Innovation
Wednesday, September 20, 2023
2:00 p.m. – 3:30 p.m.
2060 Rayburn House Office Building
Barriers to receiving high quality, comprehensive care and cures for sickle cell disease (SCD) are a significant problem in the United States, resulting in health care disparities and inequities. Individuals with SCD are living longer but unfortunately, many receive uncoordinated, inconsistent care, leading to poor clinical outcomes, avoidable complications, low quality of life, and increased costs to the healthcare system. Improving access to high quality outpatient care and to existing and forthcoming cures will improve health outcomes and quality of life for individuals with SCD.
This briefing will present an overview on the current state of health care for individuals with SCD and the critical need for comprehensive care and access to cures; an SCD warrior’s efforts to find comprehensive care; and a presentation of proposed policy strategies to improve the health outcomes of individuals with SCD. Join us to learn how you can help improve SCD care and outcomes.
