News FT-4202, FORMA’s Experimental SCD Therapy, Fares Well in Phase 1 Trial of Healthy Volunteers

News Sickle Cell Patient Describes ‘Rebirth’ After Gene Therapy

News Emmaus Partners with US Bioservices to Offer SCD Patients New Treatment Adherence Program

News FDA Grants Rare Pediatric Disease Status to Investigational Gene Therapy ARU-1801 for Sickle Cell

News GBT and Syros Partner to Develop Sickle Cell Treatments That Boost Fetal Hemoglobin

News Top 10 Sickle Cell Disease Stories of 2019

News NIH Grant Backs Project to Develop New Pain Therapy for SCD Patients

News California Earmarks $15M for Expanding Care of Adult SCD Patients

News Gene Therapy LentiGlobin Continues to Show Promising Results, Phase 1/2 Trial Data Shows

News Gene Therapy Takes Center Stage at 2019 NORD Summit

News Novartis Partners with Ghana to Treat SCD, Offering Hydroxyurea and Opening Adakveo Trial

News Adakveo Being Tested in Series of Trials, Including at Higher Dose and in Children

News HHS Secretary Alex Azar Touts White House Efforts to Fight Sickle Cell Disease

News Oxbryta to Enter ‘Confirmatory’ Trial in Sickle Cell Children in Coming Weeks, GBT Says

News FDA Approves Oxbryta, 1st Treatment Targeting Root Cause of Sickle Cell

News Hemoglobin-rich Droplets Within Blood Cells May Be Source of Sickle Shape, Study Says

News Adakveo ‘Important’ and Safe New Treatment for VOC Crises in Sickle Cell, SUSTAIN Researcher Says

News CTX001 Found to be Safe and Effective in First Tested Patient with Severe Sickle Cell Disease, Preliminary Data Suggest

News Universal Screening Tool for Socioeconomic Needs May Improve Care of Children with SCD, Study Suggests

News FDA Approves Novartis’ Adakveo, 1st Treatment for Pain Crises in Teens and Adults with SCD

News Rare Disease Film Festival Highlights Patient and Researcher Unity

News Modified CRISPR/Cas9 May Open Door to New Therapies for SCD and HIV

News Gene-Editing Tool in Ongoing Trial of BIVV003 in Treating Severe Sickle Cell Supported by Early Study

News Delta-aminolevulinate Molecule May Be Potential New Therapy for Sickle Cell Anemia, Study Suggests

News Inflammation in Airways of Sickle Cell Children Distinct from Asthma Alone, Pilot Study Suggests

News NIH and Gates Foundation Each Invests $100M to Boost Development of Affordable Gene Therapies for Sickle Cell Disease, HIV

News Enhanced Parenting Skills May Help Improve Cognitive Function in SCD Children, Study Suggests

News Caraphenol A May Facilitate Faster, More Effective Gene Therapies for Sickle Cell Anemia, Study Says

News Voxelotor May Reduce Severity and Number of Leg Ulcers, Trial Analysis Suggests

News Sickle Cell Patients’ Diet Should Be Rich in Selenium, Researchers Say

News UNITY Prenatal Test Uses Maternal Blood to Effectively Screen for SCD, Other Genetic Disorders, Study Says

News FDA Awards $2M to Phase 2 Trial of Vitamin D for Reducing Risk of SCD Respiratory Complications

News New Viral Vector May Make Gene Therapy for Sickle Cell More Effective, NIH Study Reports

News How Diet Can Help Support Sickle Cell Patients: A Nutritionist’s View

News CDC Expands SCD Data Collection Program to 7 More US States in Effort to Improve Services

News Special Program Cut Hospital Stays, Improved Well-being of Young Patients with Vaso-Occlusive Crisis, Study Finds

News NORD 2019 Rare Disease Summit Set for Oct. 21-22 in Washington, DC

News Red Cross Features Thriving Sickle Cell Patient to Increase Blood Donations, Raise Awareness

News Emergency Department Less Likely to Provide Recommended Care for VOEs, Study Finds

News ‘Sideline Sickle Cell’ Campaign Heads to Detroit to Raise Awareness about the Disease

News Hypertension Medication Valsartan May Help Prevent Vaso-Occlusive Episodes in Sickle Cell Disease, Study Suggests

News Walks, Blood Drives Among Activities Marking Sickle Cell Awareness Month

News Voxelotor Before FDA for Approval as Possible Disease-modifying Treatment for Sickle Cell

News 8th Annual SCD Therapeutics Conference Set for Sept. 10

News Sickle Cell Trait Doesn’t Predict Cognitive Decline in African Americans, Study Finds

News ‘Disorder’ Film Festival Offers Look at Siblings Touched by Rare Diseases

News At NY Genome Center, Legal Expert Presents Ethical Dilemmas in Gene Editing

News New Computer Model May Be Used to Find Medicines for Sickle Cell Disease Patients

News Nigerian-British Rugby Player Ade Adebisi Named ‘Ambassador’ for BioMedomics’ Diagnostic Tool

News ReachBio Launches Screening Platform to Find New Therapies for SCD

News Nebraska’s Neena Nizar Seeks Cure for Jansen’s, One of World’s Rarest Diseases

News ICER Will Compare Lead Candidate Therapies Crizanlizumab and Voxelotor for Efficacy, Value

News New Technology May Allow for Better Monitoring of Sickle Cell Disease

News Cognitive Decline Not Evident in Black Americans with Sickle Cell Trait, Study Finds

News Survival After Kidney Transplant Lower in SCD Patients Than Other Kidney Disease Patients, Analysis Shows

News Phase 3 Trial of Rivipansel in Treating SCD Pain Crisis Fails to Meet Goals, Pfizer Announces

News Rare Disease Groups Seek Public Support to Renew Newborn Screening Act in Senate

News Oklahoma Ranks Lowest on Programs Key to Rare Diseases on NORD’s 2019 State Report Card

News NHLBI Stepping Up Efforts to Apprise SCD Patients of Therapies and Trials

News Emmaus Takes ‘Sideline Sickle Cell’ Campaign to Annual SCD Family and Educational Event

News T-cells Promote Inflammation in Children with Sickle Cell as well as Adults, Study Finds

News $19.2M Grant to Support SCD Trial into Red Cell Exchange in US and Europe

News European Initiative Targets Diagnosis, Treatment of Rare Diseases

News FDA Grants Priority Review to Novartis’ Application for Crizanlizumab

News ‘Sickle Cell Speaks’ Campaign Raises Awareness with Aim of Eroding Stigmas, GBT and Partners Say

News Non-Invasive Prenatal Screening Test for Sickle Cell Appears Possible, Study Reports

News 45th Annual SCDAI 8K Walk, Jog, or Bike-a-Thon Set for July 20 in Chicago

News World’s First Alport Stamp Is Macedonian Mom’s Latest Win for Rare Disease Patients

News Chronic Pain in Sickle Cell Disease Underpinned by Changes in Brain Networks, Study Suggests

News Early Treatment with Hydroxyurea Lessened SCD Severity Among Infants, Study Shows

News NORD Honors Industry, Patient Advocates at Rare Impact Awards Gala

News Investigational Oral Inhibitor IMR-687 Shows Promising Results in Ongoing Phase 2 Clinical Trial

News Eurordis Unveils Integrated-care Initiative for Rare Disease Patients

News HOPE Trial Shows Voxelotor May Increase Hemoglobin, Reduce Anemia in Adults and Adolescents With SCD

News ‘Rare Barometer’ Program Helps Eurordis Shape EU Rare Disease Policy

News Howard University Symposium Focuses on Sickle Cell Cure

News Sevuparin Fails to Show Clinically Meaningful Improvements in Managing VOCs, Phase 2 Study Shows

News GBT Awards $200K in ACCEL Grants to Improve SCD Healthcare Delivery

News Sickle Cell Advocate Howard Woolley Wins Prestigious Award for Working to Improve Patient Care

News SCDAA Plans June 22 ‘Walk With the Stars’ Fundraiser in Baltimore

News FDA Grants Fast Track Status to Imara’s IMR-687 Candidate Therapy for Sickle Cell Disease

News Agency Unveils RaDaR to Help Patient Groups Develop Rare Disease Registries

News Novartis’ STEP Program Funding Projects Supporting Sickle Cell Community

News Participants Sought for Trial Evaluating Olinciguat for Sickle Cell Disease

News Stakeholders Call for Improved Care for SCD Patients in California

News Rare Diseases Constitute a ‘Public Health Issue,’ NCATS Director Warns

News SCD Experts Call for Routine Screening Among Migrants

News Cognitive Function May Be Affected in Adults with Sickle Cell Disease, Study Suggests

News CIRM and NHLBI Collaborating to Fund Cell and Gene Therapies for Sickle Cell Disease

News Findings on Abnormal Hemoglobin Assembly Could Lead to Lower-Dose SCD Medications, Researchers Say

News Holistic Treatment Approach May Help Heal Long-Lasting SCD-Related Skin Ulcers, Case Report Shows

News More Online Queries in Winter Suggest Seasonal Variations in SCD Activity

News Nonprofit Group Works to Raise Rare Disease Awareness in India

News NFL Broadcaster Huddles With Emmaus to ‘Sideline Sickle Cell’ Disease

News Loma Linda Hospital ‘Effecively Cures’ Girl Using Stem Cells Transplanted from Dad, Report Says

News Low Oxygen Regions in Brains of Sickle Cell Patients are Prone to Small Strokes, Study Shows

News Abnormal Red Blood Cells’ Shape Affect Blood Clot Formation, Study Shows

News Identifying Outcomes for Sickle Cell Disease Clinical Trials is Aim of coreSCD

News Pfizer Executive Outlines Vision for Treating Sickle Cell Disease, Other Rare Diseases

News Study Determines Low Adherence to Hydroxyurea Treatment Among Children, Adolescents