The Red Ribbon Gala and Scholarship Awards recognize those who have been strong advocates in the fight against Sickle Cell Disease and the presentation of scholarship awards to two deserving students who are living with Sickle Cell Disease and are currently in high school or attending a college of their choice.
Women with sickle cell disease have a high risk of complications during pregnancy, according to new research from French Guiana. The findings also show that women with the more severe SS-type sickle cell disease are more likely to have premature babies, and to suffer health problems themselves, than women with less severe disease types.
The study, “Pregnancy in Sickle Cell Disease Is a Very High-Risk Situation: An Observational Study,” published in the journal Obstetrics and Gynecology International, underscores that pregnant women with sickle cell disease should be supervised by a multidisciplinary team that includes primary care physicians, obstetricians, and a specialist in sickle cell disease.
Anyah Randolph, a teenager with sickle cell disease (SCD), was recently able to join her school class on a trip to Europe – and even climb the Eiffel Tower in Paris after the elevator broke – thanks to the help of hydroxyurea and Loyola Medicine pediatricians.
Anyah had a difficult childhood, as she was always in the hospital, according to her mother, Lakisia Mobley. Her life began to change after she met Loyola Medicine pediatric hematologist Dr. Natalie Kamberos, who introduced her to a new medication: hydroxyurea, a drug that keeps the blood cells round and flexible.
Dr. Kevin Williams, the Chief Medical Officer, for Pfizer’s rare disease unit, plans to help educate the masses about sickle cell disease (SCD) in a new column for the National Newspaper Publishers Association (NNPA).
Earlier this year, Pfizer Inc. and the NNPA, a trade association of more than 200 African American-owned media companies in the United States, announced a collaboration to raise awareness of SCD, a lifelong and debilitating genetic disorder that affects red blood cells. According to the Centers for Disease Control and Prevention, SCD occurs in 1 out of every 365 Black or African-American births.
Williams said that the column is a part of the collaboration between Pfizer and the NNPA to raise awareness about SCD and the importance of clinical trial participation in the process for developing new medications.
“We’ll be covering a number of topics including tips for living with sickle cell disease, common myths about the disease, and how to support a friend or family member with the disease”, said Williams.
Shakir Lateef Cannon, a 34-year-old advocate for raising awareness of sickle cell disease and improving health disparities affecting minorities, whose efforts took him to the White House in recent years, died Tuesday at Albany Medical Center Hospital, following a sudden respiratory infection, according to his mother.
The respiratory infection was a complication from the sickle cell disease he’d battled all his life, his mother, Cheryl Cannon, said. Shakir, who’d received monthly blood transfusions since having a stroke at age 3, had trouble breathing Sunday night at his home in Clifton Park and was taken by ambulance to Albany Med.
He left behind a wife of six years, Chau Duong Cannon, and their 5-year-old daughter, Kira, as well as parents, a brother and other relatives and friends.
Despite his illness, Cannon lived an active life. His mother said the monthly blood transfusions kept him from suffering in pain as badly as some other children with sickle cell disease, though in his online writings, Shakir describes having pain as an adult and also enduring not only regular transfusions but also nightly infusions to remove iron from his blood.
La Jolla Pharmaceutical company today announced the initiation of a pivotal clinical study of LJPC‑401 (synthetic human hepcidin) in patients with transfusion-dependent beta thalassemia who, despite chelation therapy, have cardiac iron levels above normal. A high level of cardiac iron puts patients at risk of cardiac complications such as heart failure and sudden death.
LJ401-BT01 is a pivotal, multinational, multicenter, randomized, controlled study that is designed to enroll approximately 100 patients across 9 countries, including the United States. Patients will be randomized 1:1 to receive either: (i) weekly subcutaneous injections of LJPC‑401, while continuing standard-of-care chelation therapy (LJPC‑401 treatment arm); or (ii) a continuation of standard-of-care chelation therapy only (observation arm).
bluebird bio said today it has acquired a manufacturing facility in Durham, NC, from an undisclosed seller for $11.5 million, with the goal of producing lentiviral vectors for the company’s cell and gene therapies.
Those therapies, bluebird said, include the cerebral adrenoleukodystrophy treatment Lenti-D, as well as LentiGlobin for transfusion-dependent β-thalassemia and severe sickle cell disease and multiple myeloma treatments bb2121 and bb21217.
bluebird said the acquisition was part of a “significant” investment in manufacturing infrastructure that includes expanding in-house expertise, creating an extensive manufacturing network, and increasing manufacturing capacity.
Designed by Marsac during her fellowship at Children's Hospital of Philadelphia, the Cellie Coping Kit was developed for children ages 6-12, and includes of a plush toy named Cellie, a deck of cards with coping and communication techniques, and a book for caregivers. The first Cellie kit was geared toward children with cancer, but has since been adapted for children with sickle cell disease, traumatic injuries, and food allergies. A kit for eosinophilic esophagitis, an allergy condition that causes inflammation of the esophagus, is in the works, as is a kit for the siblings of children with illness or disability.
The kit is designed not only to comfort children during their illness, but also to provide them with the tools they need to understand their treatment and communicate their feelings. The coping cards address various "stressors" children can face, from procedures (e.g. needle sticks), to emotional stress (anxiety and sadness), to side effects of treatment (nausea, hair loss).
This World Sickle Cell Day, the American Society of Hematology (ASH) is working on several projects to conquer sickle cell disease (SCD) worldwide. Among them, the Society has launched an effort to develop clinical practice guidelines on the management of SCD. An expansion of ASH’s commitment to identify and confront unmet needs in SCD patient care, these guidelines will be created with input from physicians and patients to address the unique needs of this underserved patient population.
Pfizer Inc. and the National Newspaper Publishers Association (NNPA), a trade association of more than 200 African-American–owned community newspapers from around the United States, are collaborating to raise awareness of sickle cell disease, a lifelong and debilitating genetic disorder that affects red blood cells.
People with rare diseases, like sickle cell disease, have unique and complex challenges. The first initiative under the collaboration will be a national poll, conducted in partnership with Howard University’s Interdisciplinary Research Team in Washington, DC. The goal is to assess the awareness of sickle cell disease, the challenges of living with the disease, and the importance of clinical trial participation in helping researchers succeed in developing potential new treatments. In a review of 174 sickle cell disease trials, difficulty enrolling patients was the stated cause in nearly half of the 30% of the trials that were terminated early.
