Updates

March 19, 2019March 19, 2019
Oral Morphine is a Viable Treatment Option for SCD Patients Experiencing VOCs, Study Says

Oral morphine treatment is a valid therapy option for patients with sickle cell disease (SCD) who are undergoing a vaso-occlusive crisis (VOC), researchers suggest. ... Read more
March 15, 2019March 14, 2019
Brexit Could Have Real Effects for UK Rare Disease Patients, Experts Warn

Madeline Collin, a 24-year-old activist with Gaucher disease, worries that patients like her will suffer deeply if Britain leaves the European Union (EU), as ... Read more
March 14, 2019March 14, 2019
Congo Delegation Visits Howard University to Bolster Collaborations on Sickle Cell Disease

A Republic of Congo delegation of health and higher education representatives recently met with Howard University officials, aiming to ultimately advance research into sickle ... Read more
March 13, 2019March 13, 2019
NIH Rare Disease Day Highlights Joint Networks Advancing Array of Research

Rare diseases affect about 30 million Americans — roughly the same number as those with type 2 diabetes. Yet only 5 percent of the ... Read more
March 12, 2019March 12, 2019
Moral Dilemmas Complicate Treatment of Rare Diseases, Says Israeli Bioethicist

With each new advance in medicine comes ethical dilemmas, from fertility treatments and newborn screening, to vaccinations, gene therapies and euthanasia. But rare diseases ... Read more

News

Weight-loss Surgery May Help Reduce VOCs in Morbidly Obese SCD Patients
March 12, 2019March 12, 2019
Bariatric surgery may help reduce the rates of vaso-occlusive crises (VOCs) in morbidly obese patients with ...
News

Women with Sickle Cell Disease May Have Earlier Decline of Ovarian Function, Study Suggests
March 7, 2019March 7, 2019
Reproductive capacity, specifically the number of eggs left in the ovaries, may decline earlier ...
News

Differences in Brain Oxygen Supply May Explain Silent Strokes in SCD Patients
March 5, 2019March 5, 2019
Brain oxygen supply is different in different regions of the brain of sickle cell ...
News

First Patient Enrolled in Phase 1/2 Trial Testing CTX001 for Severe Sickle Cell Disease
February 28, 2019February 28, 2019
A Phase 1/2 clinical trial evaluating investigational therapy CTX001 for severe sickle cell disease (SCD) has enrolled ...
News

WODC 2019 Organizers Expect 1,200 to Attend Rare Disease Conference in April
February 26, 2019February 26, 2019
The world’s biggest gathering of rare disease researchers, patient groups, pharmaceutical executives, and government ...
News

ACCEL Program Launched to Improve Healthcare Access for Sickle Cell Patients
February 21, 2019February 21, 2019
Global Blood Therapeutics (GBT) intends to improve access to high-quality healthcare for people with sickle ...

Subscribe to our mailing list

Get regular updates delivered to your email box.

Thanks! Please check your email for a confirmation email.

Vienna to Host RARE2019 Meeting on Rare Diseases
February 21, 2019February 21, 2019
About 100 scientists, researchers, pharmaceutical executives, and others will converge on Austria’s capital city ...
GBT Publishes Data from Phase 1/2 Trial of Voxelotor in SCD Patients
February 21, 2019February 26, 2019
Global Blood Therapeutics (GBT) published the results from its Phase 1/2 clinical trial that tested ...
Spectra Optia Device Save Way to Treat Sickle Cell While Using Less Blood Cell Packs, Study Finds
February 19, 2019February 19, 2019
Patients, Family and Friends to Take Part in Variety of Rare Disease Day Events Worldwide
February 14, 2019February 14, 2019
ATS Releases Guidelines for Home Oxygen Therapy in Children with Sickle Cell Disease
February 12, 2019February 19, 2019
Emmaus Unveils Co-payment Assistance Program for SCD Endari Patients
February 7, 2019February 7, 2019

FDA Revising ‘Draft Guidance’ on Developing Treatments for Rare Diseases
January 31, 2019January 31, 2019
The U.S. Food and Drug Administration(FDA) is updating its 2015 draft guidelines for drug ...
Kids with Sickle Cell Anemia are More Sedentary than Healthy Peers, Study Suggests
January 31, 2019January 31, 2019
Registration Open for Sickle Cell Foundation of Georgia’s Annual Camp New Hope
January 29, 2019January 29, 2019
Vaso-Occlusive Pain Linked to Menstruation in Some Patients, Study Finds
January 24, 2019January 27, 2019

Safety and Early Hints of Benefit Seen in Phase 1b Trial of PF-04447943
January 22, 2019January 22, 2019
Sickle cell disease (SCD) patients were safely treated with a phosphodiesterase 9A (PDE9) inhibitor ...
Howard University President Launches ‘Run To Cure Sickle Cell’ Campaign
January 18, 2019January 18, 2019
Randomized Trials Needed to Assess Benefits of Salmonella Vaccines in SCD Patients, Study Says
January 15, 2019January 15, 2019

News

Crizanlizumab Designated FDA Breakthrough Therapy for Potential in Vaso-occlusive Crisis Prevention
January 10, 2019January 11, 2019
Crizanlizumab (SEG101), Novartis‘ investigational compound for sickle cell disease (SCD), received breakthrough therapy designation from ...
News

CTX001 Earns FDA’s Fast Track Status for Treating Sickle Cell Disease
January 7, 2019January 8, 2019
The U.S. Food and Drug Administration (FDA) has awarded fast track status to investigational therapy CTX001 to treat sickle cell ...
News

Hospitals See No Link Between US Opioid Crisis and Patients’ Use of Treatment, Study Reports
January 3, 2019January 3, 2019
The U.S. opioid epidemic is not linked to higher in-hospital mortality in patients with ...
News

Rising Healthcare Costs Strain EU Budgets Even as New Therapies Flourish
December 20, 2018December 20, 2018
Cash-strapped governments across the 28-member European Union are struggling to control runaway healthcare expenditures ...
News

LentiGlobin Shows Positive Effects in Severe Sickle Cell Disease Patients, Phase 1/2 Data Reports
December 18, 2018December 18, 2018
Bluebird bio presented new positive results from the ongoing Phase 1/2 HGB-206 clinical trial evaluating ...
News

Voxelotor Can Promote Long-Term Benefits in Teens, Adults with SCD, Latest Clinical Data Show
December 11, 2018December 11, 2018
Global Blood Therapeutics’ lead therapeutic candidate voxelotor can induce rapid, robust, and sustained improvements ...
News

Delayed Hemolytic Transfusion Reaction Can Lead to Poor Outcome in SCD
December 4, 2018December 4, 2018
Hemolytic transfusion reactions, a serious complication of red blood cell transfusion, can be difficult ...
News

Novartis Announces Funding for Projects Aimed at Supporting Sickle Cell Disease Community
November 27, 2018November 27, 2018
For this year’s Solutions to Empower Patients (STEP) program, global healthcare company Novartis has ...
News

New Tool Helps Primary Care Physicians Provide Better Care for SCD Patients, Study Shows
November 20, 2018November 20, 2018
A new clinical decision support-based tool can help primary care physicians improve the care ...

October 25, 2016October 25, 2016

Kids with Sickle Cell Disease at Risk for Lead Poisoning, NYC Study Shows

News

National and local policies have led to large reductions in lead poisoning cases overall in the U.S., but severe cases are still common among children. Recently, researchers at the New York City Department of Health and Mental Hygiene (DOHMH) investigated sources of exposure, risk factors, and ... Read more

Subscribe to our mailing list

Pin It on Pinterest