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Sickle Cell Disease

All articles tagged with #sickle cell disease

Gene Therapies for Sickle Cell Disease Face Access Barriers Despite High Efficacy
health1 day ago

Gene Therapies for Sickle Cell Disease Face Access Barriers Despite High Efficacy

The FDA approved Casgevy and Lyfgenia in 2023 as the first gene therapies for severe sickle cell disease, offering potential cures with 88% to 94% efficacy. However, uptake remains low, with only 64 patients receiving Casgevy and 115 receiving Lyfgenia by late 2025. Barriers include high costs ranging from $2.2 million to $3.1 million, complex treatment processes causing infertility, and limited provider availability. While the CMS Cell and Gene Therapy Access Model aims to lower costs, uneven coverage and geographic gaps persist, leaving many eligible patients without access.

via CNBC|
#gene-therapy#health#healthcare-access
UK Teenager Achieves Functional Cure for Thalassemia via Gene Editing
health6 days ago

UK Teenager Achieves Functional Cure for Thalassemia via Gene Editing

A 16-year-old UK patient, Vian, has successfully undergone gene editing to reverse symptoms of thalassemia, eliminating the need for lifelong monthly blood transfusions. The treatment, which involves editing stem cells to produce fetal hemoglobin, is described by clinicians as a 'functional cure.' While Vian has recovered and resumed normal activities, another patient, 14-year-old JoelSamuel, is currently undergoing the same procedure for sickle cell disease, highlighting the therapy’s expanding application in UK children’s hospitals.

via BBC|
#birmingham-childrens-hospital#gene-editing#health
Illinois Expands Medical Cannabis Qualifying Conditions and Possession Rules
politics1 month ago

Illinois Expands Medical Cannabis Qualifying Conditions and Possession Rules

Illinois expanded its medical cannabis program by adding sickle cell disease and PMOS with chronic pain as qualifying conditions (bringing the total to 58). As part of SB 3222, adult‑use dispensaries can opt in to sell medical cannabis at the medical tax rate; adults may now possess up to 60 grams of flower, 10 grams of concentrates, and up to 1,000 mg of THC for medical patients. The law also doubles expungement eligibility for past convictions and tightens rules on hemp THC products. New qualifying conditions include female orgasmic disorder, endometriosis, ovarian cysts, and uterine fibroids, with Gov. Pritzker highlighting Illinois’ leadership on cannabis policy.

via Marijuana Moment|
#illinois#medical-cannabis#pmos
health-policy1 month ago

Illinois Expands Medical Cannabis Eligibility to Include PMOS and Sickle Cell Disease

IDPH adds polyendocrine metabolic ovarian syndrome (PMOS) with chronic pain and sickle cell disease to Illinois’ list of qualifying conditions for medical cannabis after review by the Medical Cannabis Advisory Board; the change, effective immediately, brings the total to 58 conditions and allows providers to certify patients right away.

via thexradio.com|
#health-policy#illinois#medical-cannabis
Gene Therapy Breakthrough Brings Hope to Black Americans Living With Sickle Cell
health3 months ago

Gene Therapy Breakthrough Brings Hope to Black Americans Living With Sickle Cell

A Louisiana man became the state’s first functionally cured sickle cell patient via 2023 FDA-approved gene therapies Casgevy and Lyfgenia; while these treatments hold promise for tens of thousands who are Black or African American, their high cost (around $2–$3 million) and limited treatment centers create access barriers, highlighting the need for broader insurance coverage, expanded care in underserved communities, and thorough discussion of risks like infertility and long‑term cancer risk.

via MS NOW|
#black-health#fda-approvals#gene-therapy
A Sickle Cell Breakthrough and the Peril of Political Attacks on Science
science3 months ago

A Sickle Cell Breakthrough and the Peril of Political Attacks on Science

A Louisiana man, Daniel Cressy, was functionally cured of sickle cell disease thanks to a breakthrough rooted in federally funded research, underscoring how modern medicine can dramatically improve lives—but the piece warns that Donald Trump’s anti-science stance could threaten such progress, which evolved from bone marrow transplants to newer therapies in the early 2010s.

via The Bulwark|
#bone-marrow-transplant#federal-funding#medical-breakthrough
Long Island Reports High Sickle Cell Burden, NYU Study Finds
health5 months ago

Long Island Reports High Sickle Cell Burden, NYU Study Finds

A NYU School of Global Public Health study of 42,200 sickle cell disease hospitalizations in New York (2009–2022) finds NYC has the most admissions, but Long Island has the highest share (10%) of hospitalizations with a major risk of death and the region incurs the highest total hospital charges. The study also shows rising rates of major severity (13% to 27%) and major mortality risk (3% to 13%), with COVID-19 likely contributing to the surge. Most hospitalized patients were Black (83%), and adults 18–29 accounted for 40%. The authors call for better access to specialists, improved pediatric-to-adult transition, and policy/funding efforts to reduce regional care gaps and emergency department burden.

via New York Post|
#health#hospitalizations#long-island
CRISPR-based sickle cell therapy earns $3M Breakthrough Prize for Thein and Orkin
health6 months ago

CRISPR-based sickle cell therapy earns $3M Breakthrough Prize for Thein and Orkin

Two researchers, Swee Lay Thein and Stuart Orkin, won the Breakthrough Prize in Life Sciences worth $3 million for work that led to Casgevy, a CRISPR-based therapy that functionally cures sickle cell disease and beta-thalassemia by disabling the BCL11A repressor to boost fetal hemoglobin. The treatment—developed with Vertex—involves editing a patient’s bone marrow cells and reinserting them, a costly, lengthy process with chemotherapy, and while a major milestone, access remains limited as scientists pursue in vivo approaches to broaden use.

via Live Science|
#bcl11a#beta-thalassemia#casgevy
Half-Matched Bone Marrow Transplant Brings Pain-Free Life to Sickle Cell Patient
health7 months ago

Half-Matched Bone Marrow Transplant Brings Pain-Free Life to Sickle Cell Patient

A woman diagnosed with sickle cell disease at 2 months old is cured after a haploidentical bone marrow transplant from her 50% genetic match at Johns Hopkins; she woke up pain-free the same day and by January 2025 was off pain medications, with doctors citing a 94% disease-free survival rate for this approach, signaling a potential cure for many sickle cell patients.

via Yahoo News Canada|
#disease-free-survival#haploidentical-bone-marrow-transplant#health
CRISPR breakthrough activates genes without DNA cuts
science9 months ago

CRISPR breakthrough activates genes without DNA cuts

Scientists at UNSW Sydney have developed a new CRISPR-based epigenetic editing technique that can turn genes on without cutting DNA, by removing chemical methyl groups that silence genes. This approach could lead to safer gene therapies for conditions like Sickle Cell disease, as it avoids the risks associated with DNA strand breaks. The research demonstrates that methylation directly controls gene activity and opens new possibilities for treating genetic disorders by reactivating silenced genes.

via ScienceDaily|
#crispr#dna-methylation#epigenetic-editing