Author Archives: Emma Day

SCDAA Response to National Academies Release of Final Report on Sickle Cell Disease and Social Security Disability Evaluations

On December 9, 2025, the National Academies of Sciences, Engineering, and Medicine (NASEM), released the second and final Sickle Cell Disease in Social Security Disability Evaluations 2025 Report. This report was completed at the request of the Social Security Administration, which tasked NASEM with reviewing the latest published research and science and producing a report on best practices and community experiences in the management and treatment of sickle cell disease. NASEM also released an interim report in June 2025.  

The Sickle Cell Disease Association of America Inc. (SCDAA), and its Medical and Research Advisory Committee (MARAC) strongly support the report’s conclusions and is eager to work with the Social Security Administration to implement appropriate and needed changes to the current Social Security disability criteria for sickle cell disease. 

This final report recognizes the broad variation in sickle cell disease and its complications as well as approaches to both acute and chronic pain management, highlighting that, for a number of reasons, pain is often managed at home or in a variety of outpatient care settings. The report’s important conclusions include: 

“There is an opportunity to improve the accuracy in the determination of disability by considering the broad variability in sickle cell disease complications and approaches to both acute and chronic pain management in a variety of settings…” 

“The frequency of sickle cell disease treatment encounters for acute complications, such as pain crises, in the emergency department and inpatient settings … is too restrictive a measure of disease severity. Growing use of alternative models of care has enabled similar levels of care in outpatient or home settings.” 

Additionally, the NASEM Report provides “overarching conclusions” related to the: 

      • full spectrum of pain and the variation in how it is experienced individuals living with SCD
      • lack of access to coordinated care
      • significant issues in transitioning from adolescence to adulthood in care, treatment and disability eligibility

The NASEM conclusions provide SCDAA and MARAC with justification to advocate for changes to the current disability criteria for sickle cell disease. 

Individuals with sickle cell disease face barriers when applying for Social Security disability and are often denied because of the overly restrictive criteria. The findings and conclusions made by this important report will enable the sickle cell disease community to initiate much needed changes.   

Sickle cell disease is a rare inherited blood disease causing red blood cells to take a sickle shape, which leads to blockages that prevent blood from reaching parts of the body. As a result, people with sickle cell complications can experience anemia, jaundice, gallstones, stroke, chronic pain, organ damage and premature death. No universal cure exists. 

Sickle Cell Disease Association of America Inc. advocates for people affected by sickle cell conditions and empowers community-based organizations to maximize quality of life and raise public consciousness while advancing the search for a universal cure. The association and more than 55 member organizations support sickle cell research, public and professional health education and patient and community services. (www.sicklecelldisease.org) 

Sickle Cell Disease is Not a Joke

This weekend’s Saturday Night Live skit about the recent historic approvals of potentially curative gene therapies for sickle cell disease is distasteful at best and harmful at worst. Earlier this month, the Food and Drug Administration approved groundbreaking new treatments that could change the lives of thousands. SNL chose to cast a spotlight on this news with a tone-deaf skit depicting a workplace Yankee Swap event in which one of the gifts is the “cure” for sickle cell disease. It is given to an African American character, who quickly trades it for a “Boogie Woogie Santa Claus” toy. The rest of the skit consists of the white characters trying to convince their two Black co-workers to choose the cure over the other Yankee Swap gifts. Their attempts are unsuccessful.

We are disappointed that Saturday Night Live chose to trivialize this landmark moment in history during their program. More than 100,000 people in the United States and millions globally are impacted by this devastating disease, and yet it is one of the few debilitating conditions that you will find people joking about on television. Earlier this year, sickle cell disease was the subject of a lame and insensitive attempt at humor on the HBO Max show Velma and, shortly thereafter, as a quasi-joke-insult by comedian D.L. Hughley on The Daily Show. Some may argue that these references are “just jokes,” but for those impacted by this disease, it is no laughing matter.

Jokes like these undermine the seriousness of this condition. Sickle cell disease (SCD) is an inherited blood disorder and rare disease that affects red blood cells. When these red blood cells become sickle-shaped, or crescent-shaped, they block blood flow to the affected part of the body, causing irreversible organ and tissue damage. When this happens, individuals with sickle cell can suffer from intractable, crippling acute pain called a “crisis” and are at elevated risk for strokes, damage to affected tissue, and all too often, an early death. In fact, a recent study showed the median age of death of those suffering from chronic sickle disease complications was only 43 years.

SNL’s treatment of race in the Yankee Swap skit also misses the mark. Part of the “humor” revolves around the common myth that only Black people can have sickle cell disease. While it does disproportionately impact the Black community, sickle cell does not discriminate. People of all ethnic backgrounds can inherit the disease. In the United States, Hispanic and Latino populations have the second highest incidence, but Asian, Indian, Native American and – yes – White people, can all be born with the disease. On a global scale, sickle cell disease affects people from countries around the world, including Italy, India, the United Kingdom and Jamaica. One doesn’t develop sickle cell disease, nor can one “catch it.” Individuals are born with it, and there is no universal cure.

Why are we joking about a disease as serious as this one? Many people don’t understand the devastating reality of the condition. The onset of sickle cell pain is sudden and debilitating. A pain crisis is relentless and can last for hours or for days. It has been described as feeling like you are walking on hot coals or like shards of glass are traveling through your veins. Far too often, when individuals living with sickle cell disease, or “warriors” as they call themselves, are in crisis and seek medical care in some emergency departments, they face long waiting periods, are accused of exaggerating symptoms for attention, and far, far too often are characterized and treated as if they are drug seekers.

For physicians who are knowledgeable about sickle cell disease and experienced in caring for those living with it, their ability to prescribe the very drugs that will help their patients is hampered by current federal regulations put in place to address the opioid crisis thus limiting how these drugs can be used in cases such as sickle cell. Layer on issues of health care inequity, discrimination and limited access to consistent, comprehensive quality care and the word “crisis” takes on new meaning.

Community-based organizations, such as the 50-plus members of the Sickle Cell Disease Association of America, Inc., spanning 29 states, are on the ground and focused on providing support, resources, and services to serve more than 500,000 children and adults living with or impacted by sickle cell disease.

Sickle cell disease also puts a strain on caregivers and family members, who must fit trips to the emergency room, doctors’ appointments and sick days into the rigors of daily life. Parents of children with sickle cell may lose wages, promotion opportunities or jobs as they try to support their family while attending to pain crises and their child’s care. This pressure can cause personal and professional instability, compromise mental health and wellness, and, in too many cases we have seen, lead to homelessness.

It is for all of the above, and more, that the Sickle Cell Disease Association of America, Inc., condemns the use of sickle cell disease as a punchline. It demeans and ridicules a condition that people are born with and from which they will face devastating health challenges throughout their lifetimes.

Stereotypes and misinformation reinforced by thoughtless comedy have real-life consequences. Sickle cell patients struggle daily to be taken seriously—in school, at work and even playing sports. As we work to change the perception of sickle cell and increase education surrounding this condition, insensitive and inappropriate jokes like these demean, marginalize and disrespect those living with the disease. They work against progress and contribute to the spread of misinformation. As a society, we must do better and treat rare diseases and the people who live with them with the respect they deserve.

Sickle cell is not a joke.

Gene Therapy: What You Need to Know (Warrior FAQs)

Two gene therapies were recently approved by the Food and Drug Administration (FDA) to treat sickle cell disease: Casegevy from CRISPR/Vertex and Lyfgenia from bluebird bio. You probably have questions about these new treatment options. Read more below.

Is gene therapy a cure for sickle cell disease?
Gene therapy is a potentially curative therapy. This means that it could act as a cure, but it is too new to say for sure. It causes a big decline in pain episodes, but we need to learn more about long-term impacts and side effects. It is also not a “one-and-done” treatment. The FDA currently recommends 15 years of patient follow up.

How does gene therapy work?

When will it be available?
Likely in early 2024.

Am I eligible for gene therapy?
Casgevy and Lyfgenia are approved for people ages 12 and up. Sickle cell disease SS and S-beta-zero-thalassemia are eligible. The FDA indicates that sickle cell disease SC is not included. Additionally, you may also not be able to receive gene therapy if you have:

  • A recurring viral infection
  • Significant organ damage

Additionally, if you have a matched sibling, you should go down the path of a matched-sibling-donor bone marrow transplant instead of gene therapy. Talk to your doctor about this option.

What are the side effects?
Gene therapy requires you to have chemotherapy. This means it could result in:

  • Infertility or secondary cancer
  • Temporary weakening of the immune system so that you cannot fight off any infections
  • Temporary hair loss

Where can I receive gene therapy?
Treatment will likely be at an existing bone marrow transplant center that also works with sickle cell disease experts. These may be hard to find. SCDAA will be providing a list of facilities, once identified, on our website: sicklecelldisease.org.

How much will it cost? Will insurance cover it?
Gene therapy is expensive, and FDA-approved high-cost medications can come with barriers. Casgevy is estimated to cost $2.2 million, and Lyfgenia is estimated to cost $3.1 million. We are still waiting to hear how insurance companies will handle gene therapies.

Does gene therapy work for all types of SCD?
As far as we know, yes. It is designed to be able to help raise fetal hemoglobin (HbF), which should work for all different kinds of sickle cell disease. However, the amount of experience with the different kinds has not been nearly the same – we know the most for SS and S Beta zero thalassemia types.

Are we the first community to receive gene therapy?
Casgevy is the first approved use of gene editing. However, gene addition therapy has been used to treat other conditions, including:

  • Retinal degeneration
  • Spinal muscular atrophy
  • Beta-thalassemia
  • X-linked Adrenoleukodystrophy
  • Hemophilia A & B
  • Bladder cancer
  • Acute-lymphoblastic leukemia

To learn more about the gene therapies used to treat these conditions, click here.

For a longer (but not complete) list of conditions that have been treated using gene therapy, click here.

Is it safe? How do I know if this is right for me?
For many people, the benefits of this new treatment outweigh the risks. Your doctors will help you determine whether this is a good option for you.

What questions should I ask my doctor?

  • How long will this take?
  • What is the time commitment?
  • Where is the nearest treatment center?
  • What are my other options?

How do I learn more about gene therapy?
There are several resources available. The below sources are considered trustworthy and non-biased by SCDAA.

To learn more about Vertex’s Casgevy, visit casgevy.com. To learn more about bluebird bio’s Lyfgenia, visit my bluebird support.

We encourage you to subscribe to our email list for news and updates.

Updated Dec. 14, 2023, at 11:09 a.m. EST

Please note: A previous version of this FAQ incorrectly stated that Casgevy is approved for people ages 12 and up and Lyfgenia is approved for those ages 12 to 50. This statement has been corrected to note that both Casgevy and Lyfgenia are approved for people ages 12 and up.

Gene Therapy is Approved!

We are very excited to share that today, Dec. 8, the Food and Drug Administration approved two gene therapies to treat sickle cell disease! These potentially curative therapies are the first treatments of their kind available to individuals with SCD. We are heartened by this approval and are proud to support our community during this milestone moment. SCDAA will be releasing a full statement and additional information for patients and caregivers soon. To learn more about these approvals, click here.

 

 

CDC SCD Pregnancy Fact Sheets

Learn more about how to stay healthy leading up to, during and after a pregnancy with these newly developed fact sheets from the Centers for Disease Control and Prevention (CDC), the Foundation for Women & Girls with Blood Disorders, the American Society of Hematology and the Sickle Cell Reproductive Health Education Directive.

NOW IN SPANISH!

 

In Memory of Dr. Lennette Benjamin

The Sickle Cell Disease Association of America, Inc., (SCDAA) is saddened to hear the news of the passing of Dr. Lennette Benjamin. Dr. Benjamin was a trailblazing physician who made many outstanding contributions to the sickle cell community. She was one of the first to establish a “day hospital” as an alternative to the emergency room for pain management – an approach that is today recognized as a best practice in care.  

Prior to her retirement, Dr. Benjamin led the Montefiore Sickle Cell Center for Adults in the Bronx, New York. She was an SCDAA board member emeritus and served on the SCDAA Medical and Research Advisory Committee. 

Dr. Benjamin was also a dedicated advocate and mentor. She made a global impact and raised sickle cell awareness in West Africa, Brazil and beyond. “She was always looking out for others, patients and peers alike,” recalls Dr. Lewis Hsu, SCDAA chief medical officer. “She was full of warm advice and expert guidance.”

Dr. Benjamin passed away in Houston, Texas on Oct. 20 at the age of 82. To share your sympathies, please click here.

SCDAA’s National Office and Board of Directors pay tribute to Dr. Benjamin’s outstanding life and career and send our deepest condolences to her family, friends and loved ones. 

CMO Speaks: Making ED Care Better for SCD – Progress in 2023

Welcome to CMO Speaks, a blog featuring the voices of SCDAA’s clinical leadership team. This article was written by Dr. Lewis Hsu, SCDAA’s chief medical officer.

The Emergency Department is no one’s favorite place, but unfortunately individuals with sickle cell disease may pay it frequent visits for emergency care. Encountering problems in ED care is distressingly common. During the SCDAA Annual National Convention in October 2023, SCDAA participated in a joint session with the Emergency Department Sickle Cell Care Coalition (EDSC3) entitled “Summit on Emergency Department Sickle Cell Care.”

The special morning session, Frameworks to Improve the Emergency Department Experience for Sickle Cell Disease, focused on existing guidelines and efforts related to dissemination and education of emergency medicine clinicians. The keynote speaker for this morning session was Dr. Aisha Terry, president of the American College of Emergency Physicians. Videos expressing the views of people living with sickle cell disease and those that care for them give further insight into the current state of care delivered in the emergency department.

Other speakers included:

  • Dr. Henry Young: Point-of-Care Tool to guide management of SCD
  • Dr. Paula Tanabe: Statewide standardization of care processes, the North Carolina experience
  • Dr. David Brousseau: Implementation through a research network, the PECARN experience
  • Yvonne Carroll: IASCNAPA sickle cell disease bootcamp for nurses

Major Takeaways on ED Care from the Convention

  1. Dr. Paula Tanabe made presentations that included implementation tips for Individualized Pain Plans (IPP) to help ED doctors and nurses to see that the IPP can help speed up their workflow. She suggested that providers put the IPP in the health record where a Patient Portal can access it. One individual reportedly showed their IPP to a new ED when traveling out of state, and that ED acted upon the IPP as credible information.
  2. SCDAA is partnering with MedicAlert to store IPPs online and provide patients with a bracelet/wallet card with a QR code that links to their information. We hope that the MedicAlert imprint will make the IPP credible.
  3. The ACEP Point-of-Care Tool for sickle cell (website and mobile app) built into their algorithm for sickle cell acute management that dosing of pain meds should be guided by IPP as first choice, weight-based dosing as second choice.
  4. A new calculator for sickle cell experts to create IPP dosing in the ED based upon the individual’s chronic opioid intake was created by Dr. Paula Tanabe and Dr. Patricia Kavanagh. The tool was validated in a clinical trial COMPARE-VOE. The Excel calculator and instructions are posted for health care providers to use at the National Alliance of Sickle Cell Centers website.

Lewis Hsu, MD, PhD, is a pediatric hematologist who serves as director of the Sickle Cell Center and professor of pediatrics for the University of Illinois at Chicago. He has conducted sickle cell research, published over 100 publications and co-authored “Hope and Destiny: The Patient and Parent’s Guide to Sickle Cell Disease and Sickle Cell Trait.” He currently serves as the SCDAA Chief Medical Officer. 

New Opiate Dosing Calculator for Health Care Providers

NEW RESOURCE AVAILABLE: This tool from the National Alliance of Sickle Cell Centers can help sickle cell providers make important decisions about dosage when prescribing opiates. The calculator was developed was Paula Tanabe, RN, Ph.D., and Patricia Kavanagh, M.D., through a grant funded by the National Heart, Lung and Blood Institute. Click here to access the resource.

Click here to read the full study.  

CMO Speaks: Gene Therapy for SCD (Part 2)

Welcome to CMO Speaks, a blog featuring the voices of SCDAA’s clinical leadership team. This is part two of a three-part series on gene therapy for sickle cell disease. Part two was written by Dr. Lewis Hsu.

What comes up for you when you hear the words “gene therapy?” If it makes you feel overwhelmed, skeptical and/or confused, you’re not alone. There are a lot of misconceptions about gene therapy, and it can be hard to understand why it’s so important. Get some answers to frequently asked questions about gene therapy below!

Q: Is gene therapy for sickle cell disease a surgery? How long does it take?
A: It is not a surgery, more of a process. It can take a while to complete. We advise people considering gene therapy to estimate that it will be like a full-time job for the first six months and continue to be very time intensive for one to two years afterward.

Q: Where can I get information about gene therapy for sickle cell disease?
A: We encourage you to start with these online resources:

New updates are presented at sickle cell conferences annually. Consider registering for the SCDAA Annual National Convention, the Foundation for Sickle Cell Disease Research’s Annual Sickle Cell Disease Research and Educational Symposium and others.

Q: How do I sign up for gene therapy?

A: There are at least five groups trying to develop gene therapy for SCD. All are currently conducting clinical research studies. The specialized team that does gene therapy overlaps with the team preforming bone marrow transplants. You can search for research opportunities using SCDAA’s Clinical Trial Finder or by visiting ClinicalTrials.gov. Signing up for gene therapy requires a lot of discussion and deep thought by the patient and their whole family. Read this statement from SCDAA’s Medical and Research Advisory Committee to learn more about what it means to participate in a clinical trial.

Q:  What connects sickle cell disease to gene therapy?

A:  Sickle cell disease is inherited, so if a person’s bone marrow stem cells have the genes for sickle hemoglobin, the red blood cells will cause sickle cell disease (SCD). Medicines can make SCD symptoms milder, but they cannot stop a person’s body from producing sickle hemoglobin. One way to stop the process and “cure” the disease would be through a stem cell transplant. However, this involves finding a donor – which can be tricky. The ultimate way to cure a genetic disease like sickle cell is by adjusting the genes so they do not make sickle hemoglobin in the first place – that is what we mean when we talk about gene therapy. It is a long-term cure that doesn’t require a donor. There are several techniques to do gene therapy.

Q:  What was the recent news about gene therapy and sickle cell disease?

A:  There was a lot of news in January – March 2023!

  1. The New York Times published this article and National Public Radio reported that gene therapy is a pioneering treatment for sickle cell disease. They highlighted the voices and experiences of advocates from around our community.
  2. Two pharmaceutical companies (bluebird bio and Vertex/CRISPR) announced that they will bring their SCD gene therapy clinical research results to the U.S. Food and Drug Administration (FDA) for possible approval as safe and effective in the first quarter of 2023. In late February, three other groups (Sangamo, Graphite Bio, Intellia) each announced the closure of their SCD gene therapy clinical research studies, apparently as business decisions.
  3. The Third International Summit on Human Genome Editing was held in London on March 6-8, 2023, and a lot of time was devoted to discussing sickle cell disease, including science, ethics and patient perspectives. One perspective is that gene therapy is one of the greatest advancements for sickle cell disease and provides hope for a “universal cure.” Victoria Gray, the first person to be cured of SCD using the CRISPR genome editing technology, was a special featured speaker. She told a powerful story about her life before and after the gene therapy. SCDAA board member Dr. Melissa Creary spoke as someone contemplating gene therapy and shared her concerns about equity and ethics related to the technology.

Q: Is the sickle cell community in favor of gene therapy?

A: Yes, gene therapy potentially could cure anybody with SCD and lead us to a universal cure. However, many important details need to be worked out before we reach that point.

Q: Would this be funded by taxpayer money? Why should a society pay the high cost of gene therapy? Or the high costs of any SCD care?

A: The high upfront cost will have a “return on investment” in the long term. Direct savings will be seen in lower medical costs for the rest of our lifetimes. Indirect economic benefits to society include fewer absences from work for individuals and caregivers, less suffering, more productive taxpayers and less crowded hospitals and emergency departments. We lack actual long-term data, but math models could estimate a “return on investment.”

Q: Does everybody with sickle cell disease want gene therapy?

A: Not yet. Some worry about side effects of gene therapy preparation, including the risks of infertility and unknown risks of leukemia. Others cannot devote the time (a year or more) for the gene therapy process. Some people feel that their current medications are controlling their SCD quite well and don’t feel the need to change their treatment plan. Some have misinformation about the process and might change their decision with better awareness. Some people simply want to “wait and see” about any new medical treatment.

Q: Will health disparities limit access to gene therapy?

A: Inequities stemming from racism, stigma and poverty are a big problem in sickle cell care now. Unless proactive steps change the US health care system, gene therapy will magnify these inequities in the US. The global impacts of gene therapy must be considered as well. Can the vast population with SCD in sub-Saharan Africa and elsewhere get access to gene therapy, or will Americans reap the greatest benefits?

Q: Should all government supported funds for sickle cell disease go to researching gene therapy?

A: It should not be an “all-or-nothing” decision. There can be a “return on investment” for federal and state funding to fulfill the evidence-based standards of care for SCD. Better care for pain in the emergency department, more access to FDA-approved medications for SCD, more comprehensive care centers with staff who are trained in modern guidelines and more awareness of SCD and sickle cell trait are all high priorities for the SCD community. People should not continue to suffer substandard SCD care while waiting for gene therapy to be developed.

If I have 25 seconds to comment on sickle cell disease gene therapy:

  1. Gene therapy progress is accelerating in SCD (“hitting the gas”). bluebird bio and Vertex/CRISPR are bringing data to FDA to ask for approval.
  2. The high upfront cost of SCD gene therapy will be worthwhile because of the “return on investment.”
  3. Individuals with SCD should not continue to suffer substandard care while waiting for gene therapy to be developed.

https://youtu.be/DqETbmgFdto

 

Lewis Hsu, MD, PhD, is a pediatric hematologist who serves as director of the Sickle Cell Center and professor of pediatrics for the University of Illinois at Chicago. He has conducted sickle cell research, published over 50 peer-reviewed papers and co-authored “Hope and Destiny: The Patient and Parent’s Guide to Sickle Cell Disease and Sickle Cell Trait.” He currently serves as the SCDAA Chief Medical Officer.