News Crizanlizumab Lowers Pain Crises in At-risk Sickle Cell Patients, Ad-hoc Trial Data Show

News Multi-Organization Partnership to Encourage Blood Donation and SCD Awareness

News Agreement With Cardinal Health Widens Distribution of Endari for Emmaus

News Advocacy Groups, Doctors Question Rising Prices of Rare Disease Treatments

News #NORDSummit – More Than 700 Expected to Attend Oct. 15-16 Rare Disease Summit in Washington

News IP-10 Could Be Biomarker of Inflammation in Pregnant Sickle Cell Patients, Study Reports

News Sickle Cell Patients in UK Survey, Especially Those 16 to 20, Voice Problems with Care and Pain Relief Given

News Activities Underway in US for Sickle Cell Disease Awareness Month

News GlycA Seen as Not a Good Indicator of Inflammation in SCD Patients, Study Shows

News Altemia Shows Promising Results in Phase 2 Trial for SCD Children

News Emmaus Life Sciences Expands Distribution of Sickle Cell Disease Therapy, Endari

News Roche Sells Cardio Antibody to GBT for Vaso-Occlusive Crises in SCD

News Siklos, Approved for Children with Sickle Cell Anemia, Now Available in US Pharmacies

News NIH Awards $4 Million for Research on Inhaled Corticosteroids for SCD

News Sickle Cell Treatments Can Destroy Germ Cells in Boys, Affecting Fertility in Adulthood, Study Suggests

News Approval of Sickle Cell Disease Bill by Senate Committee Welcomed by Emmaus Life Sciences

News Endari Will Soon Be Available to Sickle Cell Patients in the US Military

News HRI Protein Identified as Possible New Therapeutic Target for SCD, Study Shows

News Endari Reduces Pain Crises, Hospitalizations in Sickle Cell Patients, Phase 3 Trial Shows

News Blood Tests and Funding Can Save African Lives, Especially Children, Official Says

News White Blood Cells Regulate Levels of Fetal Hemoglobin in Sickle Cell Patients, Study Shows

News Researchers Raise Funds for Phase 1 Trial to Test Medical Cannabis in Sickle Cell Disease

News Music Therapy Helps Relieve Pain in Adults with Sickle Cell Disease, Pilot Trial Suggests

News Kids with Sickle Cell Disease and Sleep Apnea at Higher Risk of Complications, Study Finds

News First Results from Phase 3 Trial Expected to Support FDA’s Accelerated Approval of Voxelotor for Sickle Cell

News Gene Therapy LentiGlobin Continues to Show Good Results, Bluebird Bio Says

News Sickle Cell Disease Association and Emmaus Partner to Increase Education, Awareness

News Sickle Cell Disease Association Launches First Patient-powered Registry

News MaxCyte and NIH Office Partner to Develop Cell Therapy for Sickle Cell Disease

News Potential Treatment Targeting Nitric Oxide Levels, Olinciguat, Named Orphan Drug by FDA

News Platform to Diagnose, Monitor SCD Wins Vodafone Wireless Innovation Project Grant

News Potential Sickle Cell Treatment Altemia Shows Promise in Phase 2 Trial, Data Reports

News FDA Places Clinical Hold on IND Application for CTX001 for Sickle Cell Disease

News US ‘Right to Try’ Law Meets with Mix of Praise and Criticism, Including Among Those with Rare Diseases

News Loss of Cell Defense Mechanism Worsens Sickle Cell Disease Severity, Mouse Study Shows

News Kids with Sickle Cell Disease Aren’t Receiving Key Vaccines, Michigan Study Finds

News #ERDC2018 – When Treating Rare Disease Patients, Don’t Overlook Quality of Life, Panel Urges

News Mutation Associated with Sickle Cell Disease Corrected in Patient’s Stem Cells, MaxCyte Reports

News Sickle Cell Trait Not Associated with Increased Stroke Risk, Study Suggests

News ‘Rare Impact Awards’ Dinner Marks Orphan Drug Act and NORD at 35

News #ECRD2018 – EU Must Do More for Rare Disease Patients, Eurordis Leaders Say

News FDA Accepts Collaborative IND Application for BIVV003 to Treat Sickle Cell Disease

News Stigmatizing Language in Medical Records Affects Patient Care, Study Shows

News St. Jude Hospital Given $1M Grant from Links Foundation to Advance 3 Sickle Cell Disease Programs

News Stem Cells from Half-Matched Donors in New Approach Reported to Cure 7 Sickle Cell Patients

News #ECRD2018 – Patient Access and Collaboration Focus of Eurordis Meeting May 10-12

News Sickle Cell Groups, Pfizer Work to Bring African-Americans into Clinical Trials

News Human Genome Project Team at NIH Honored for Advancing Research into Genetic Diseases

News In-person Caregiver Education Increases Sickle Cell Trait Knowledge, Study Reports

News Sickle Cell Disease Researchers Identify a Children’s Stroke Prediction Tool

News Novo Nordisk Obtains Worldwide Rights to EpiDestiny’s Sickle Cell Disease Therapy

News NIH Agency Pioneers Collaborative Research into Rare Diseases

News Researchers ID Specific Genetic Marker That Can Lead to SCD Complications

News Altemia Granted Orphan Drug Status in EU as Potential Therapy for Pediatric Sickle Cell Disease

News Gene Editing Tool May Be Key to New Therapies for Sickle Cell Anemia, Study Reports

News Genome Sequencing of Children With Unexplained Symptoms May Help ID Sickle Cell Cases, Study Shows

News Inhaled Corticosteroids Don’t Seem to Benefit SCD Kids with Acute Chest Syndrome, Study Says

News Sickle Cell Patients at Higher Risk for ER Readmission, Hospitalization, Study Finds

News Racial Bias a Reality for Young Patients with Sickle Cell Disease, Study Shows

News Researchers Report Great Success in Stemming Graft-Versus-Host Disease in SCD Patients

News Genome Sequencing and Its Clinical Potential Focus of NYC Rare Disease Day Event

News European Regulators Ready to Review Emmaus’s Sickle Cell Disease Therapy

News ‘A Disease May Be Rare, Hope Should Never Be,’ Says Boy with SMARD at Connecticut Rare Disease Day Event

News Rare Disease Groups Welcome FDA’s Embrace of ‘Real World’ Data in Clinical Trials

News CRISPR/Cas9 Gene Editing Shows Potential to Treat Sickle Cell Disease, Study Reports

News US Lawmakers Urge Bipartisan Support for Rare Disease Research, Patients’ Needs

News Phase 1 Clinical Trial for Novel Sickle Cell Transplant Procedure Receives $5.74 Million

News Retrophin, Horizon Donate $3M Each to Rare Disease Institute at Children’s National

News Gamida Presents Updated Data from Phase 1/2 Trial on Experimental Sickle Cell Treatment

News BioNews to Cover 3 Rare Disease Day Events, Including NIH Conference

News Patient Advocacy Groups Worldwide Plan Events to Mark Rare Disease Day, Feb. 28

News Antibiotic Preventive Treatment Rates for IPD Very Low Among Sickle Cell Children, Study Says

News Imara Begins Phase 2 Sickle Cell Disease Trial Testing IMR-687 in Adults

News Rare Disease Groups, Patients Differ on ‘Right to Try’ Bill Before US Congress

News Hypersensitivity to Allergens May Increase Risk of Acute Chest Syndrome in Sickle Cell Anemia Children

News Two Types of CRISPR Gene Editing Could Be Useful in Treating Sickle Cell Anemia, Editas Reports

News Hydroxyurea Treatment in Men with SCA Leads to Drop in Total Sperm Count, Study Shows

News Medicaid to Cover Endari for Sickle Cell Disease in the U.S.

News NORD Fundraiser Sets 7,000-Mile ‘Bike, Run or Walk’ Goal for 7,000 Rare Diseases

News How a Man and Woman, Both with a Sickle Cell Anemia Mutation, Had a Healthy Child

News Entrepreneur to Design New Voice-recognition Mobile App for Monitoring Blood Disease

News Endari Now Available by Prescription in US for Sickle Cell Disease Treatment

News FDA Grants Breakthrough Therapy Designation to Voxelotor

News Emmaus Helping Sickle Cell Disease Patients Outside US Obtain Early Access to Its Therapy

News Bluebird Bio Presents Positive Early Results of Gene Therapy LentiGlobin for Sickle Cell Disease

News Novartis Therapy Crizanlizumab Prolongs Time to First Sickle Cell Pain Crisis, Study Finds

News Voxelotor Improves Sickle Cell Disease of 83% of Adolescents in Phase 2a Trial

News Prolong Completes Enrolling Patients in Phase 2 Trial of Sickle Cell Disease Therapy Sanguinate

News Increasing Doses of Hydroxyurea Benefits Young Sickle Cell Patients

News CTX001 to Be Developed as Gene-Editing Therapy for Sickle Cell Disease

News Intellia Uses Gene Editing to Replace Defective Blood Component in Sickle Cell Disease

News Sickle Cell Disease Patients Use Opioids More When Experiencing Negative Emotions, Study Finds

News New Online Platform Created to Support Sickle Cell Disease Community

News Teen with Sickle Cell Disease Takes Class Trip to Europe with Loyola Medicine’s Help

News Expert Analysis Updates Advice on Managing Sickle Cell Retinopathy

News Review Board Finds Global Blood’s Sickle Cell Disease Therapy Voxelotor Is Safe

News Rare Disease Patient Groups Unite to Preserve Orphan Drug Tax Credit in US

News Increase in a Type of Gut Bacteria Linked to More Pain in Patients with Sickle Cell Disease

News Sickle Cell Patient with Severe Anemia Rapidly Improves with Voxelotor, Case Study Shows

News Alzheimer’s Treatment Memantine Shows Promise in Treating Sickle Cell Disease