Stuart Orkin
Dr. Stuart Orkin didn’t set out to develop a historic treatment for sickle cell disease 45 years ago when he decided to study how blood cells formed. He became a researcher at Harvard Medical School just as scientists learned how to clone, or make copies of, genes. “Everybody was talking about how we could now fix genetic diseases,” he says. “But no one had any clue what that really meant; it was kind of a Disney fantasy.” Orkin focused his attention on the hemoglobin gene, which is mutated in people with sickle cell anemia and another set of blood diseases called beta thalassemia. Understandi...
Source: TIME: Health - May 2, 2024 Category: Consumer Health News Authors: Alice Park Tags: Uncategorized Accolades franchise Magazine Special Project sponsorshipblock time100health2024 Source Type: news
Exa-Cel Eliminates Vaso-Occlusive Crises in Most Sickle Cell Patients
THURSDAY, April 25, 2024 -- For almost all patients with sickle cell disease, treatment with exagamglogene autotemcel (exa-cel) eliminates vaso-occlusive crises, according to a study published in the April 24 issue of the New England Journal of... (Source: Drugs.com - Pharma News)
Source: Drugs.com - Pharma News - April 25, 2024 Category: Pharmaceuticals Source Type: news
Researchers publish final results of key clinical trial for gene therapy for sickle cell disease
In a landmark study, an international consortium led by researchers at Children's Hospital of Philadelphia (CHOP) published the final results of a key clinical trial of the gene therapy CASGEVY (exagamglogene autotemcel) for the treatment of sickle cell disease in patients 12 years and older with recurrent vaso-occlusive crises (VOCs). The study found that 96.7% of patients in the study did not have any vaso-occlusive crises (VOCs) - a blockage that results in lack of oxygen and painful episodes - for at least one year, and 100% were able to remain hospitalization-free for the same length of time. (Source: World Pharma News)
Source: World Pharma News - April 24, 2024 Category: Pharmaceuticals Tags: Featured Research Research and Development Source Type: news
Suffering Shouldn ’ t Be a Normal Part of Womanhood
One of the most important lessons medical schools teach is one my mom mastered as a teenager left to fill her own mother’s shoes: how to figure out who is really sick and needs immediate attention and who can wait (or what we in the medical field call “triage”). Nothing I learned in med school or since has contradicted what I learned at Bertha’s knee.
At the time, the practice of medicine was rudimentary—and that’s putting it nicely. So, the diagnoses typically made at home were probably not that different from those of a bona fide doctor. The more serious common ailments were things ...
Source: TIME: Health - April 9, 2024 Category: Consumer Health News Authors: Dr. Sharon Malone Tags: Uncategorized health Source Type: news
FDA Approves Xromi (hydroxyurea) Oral Solution for Use in Pediatric Patients with Sickle Cell Anemia
April 4, 2024 -- The U.S. Food and Drug Administration (FDA) has approved Xromi, an oral solution formulation of hydroxyurea indicated to reduce the frequency of painful crises and reduce the need for blood transfusions in pediatric patients aged 6... (Source: Drugs.com - New Drug Approvals)
Source: Drugs.com - New Drug Approvals - April 4, 2024 Category: Drugs & Pharmacology Source Type: news
Crude Sickle Cell Birth Prevalence 28.54 per 10,000 in Non-Hispanic Blacks
MONDAY, April 1, 2024 -- The crude sickle cell disease (SCD) birth prevalence is 28.54 per 10,000 non-Hispanic Black newborns, according to research published in the March 28 issue of the U.S. Centers for Disease Control and Prevention Morbidity and... (Source: Drugs.com - Pharma News)
Source: Drugs.com - Pharma News - April 1, 2024 Category: Pharmaceuticals Source Type: news
Pfizer puts 91,000 square feet on sublease market following job cuts
Pfizer bought the South San Francisco company, which developed an approved sickle cell disease treatment, in fall 2022 for $5.4 billion. (Source: bizjournals.com Health Care:Pharmaceuticals headlines)
Source: bizjournals.com Health Care:Pharmaceuticals headlines - March 28, 2024 Category: Pharmaceuticals Authors: Ron Leuty Source Type: news
Birth Prevalence of Sickle Cell Disease and County-Level Social Vulnerability - Sickle Cell Data Collection Program, 11 States, 2016-2020
This report describes the prevalence of sickle cell disease among newborns born to mothers in counties with high social vulnerability. (Source: CDC Morbidity and Mortality Weekly Report)
Source: CDC Morbidity and Mortality Weekly Report - March 28, 2024 Category: American Health Tags: Health Equity MMWR Morbidity & Mortality Weekly Report Social Determinants of Health Source Type: news
Many Children With Sickle Cell Anemia Do Not Meet Quality Indicators
MONDAY, March 18, 2024 -- A number of children with sickle cell anemia (SCA) have low rates of receipt of recommended antibiotic prophylaxis and annual transcranial Doppler (TCD) ultrasound, according to a study published online March 6 in... (Source: Drugs.com - Pharma News)
Source: Drugs.com - Pharma News - March 18, 2024 Category: Pharmaceuticals Source Type: news
Annual Indirect Economic Burden of Sickle Cell Disease Over $2 Million
WEDNESDAY, March 13, 2024 -- Adults with sickle cell disease (SCD) are more likely to report employment loss, and caregivers of children with SCD report more missed days of work, according to a study published online Feb. 29 in Blood... (Source: Drugs.com - Pharma News)
Source: Drugs.com - Pharma News - March 13, 2024 Category: Pharmaceuticals Source Type: news
Sickle Cell Pain Hospitalizations Rose After CDC's Opioid Recs
(MedPage Today) -- A downward trend in opioid prescribing practices for patients with sickle cell disease (SCD) followed the CDC's 2016 opioid guidelines for chronic pain, an analysis of claims data showed, but so did a steady increase in pain... (Source: MedPage Today Hematology/Oncology)
Source: MedPage Today Hematology/Oncology - March 11, 2024 Category: Hematology Source Type: news
IVF gave hope to patients trying to build their families. The turmoil in Alabama put that in doubt
Women over 35 and those facing serious diseases like cancer, lupus and sickle cell are among the most likely to turn to IVF to build the families they desperately want (Source: ABC News: Health)
Source: ABC News: Health - March 5, 2024 Category: Consumer Health News Tags: Health Source Type: news
New sickle cell gene therapies are a breakthrough, but solving how to pay their high prices is a struggle
The approval of two gene therapies to treat sickle cell disease has given hope to patients who suffer from the debilitating disease, which overwhelmingly affects Black people and people of color. Health officials now face a challenge in finding a way to provide equitable access to the costly…#michaelgoodwin #bluebirdbio #lyfgenia #juliekanter #adultsickleclinic #medicaid #katemcevoy #vertex #fda #merck (Source: Reuters: Health)
Source: Reuters: Health - February 24, 2024 Category: Consumer Health News Source Type: news
Progress, Not Cure, for Sickle Cell Disease, Says Ethicist Progress, Not Cure, for Sickle Cell Disease, Says Ethicist
Art Caplan discusses progress made for treating sickle cell disease and the importance of not labeling it as a cure.Medscape Business of Medicine (Source: Medscape Today Headlines)
Source: Medscape Today Headlines - February 22, 2024 Category: Consumer Health News Tags: Family Medicine/Primary Care Commentary Source Type: news
Crispr Reports Its First Profit In 10 Quarters; Shares Zoom Higher
reported its first profit in 10 quarters on Wednesday, and CRSP stock surged out of a buy zone. This is the company's first earnings report following the historic approval of Casgevy, its gene-edited treatment for sickle cell disease and beta thalassemia. Crispr hasn't yet notched any sales of…#crsp #marketsmithcom #allisongatlin #ibdagatlin #medtronicstockyoyoes #trackandbuy #avoidbiglosses (Source: Reuters: Health)
Source: Reuters: Health - February 21, 2024 Category: Consumer Health News Source Type: news
