Updates

February 14, 2019February 14, 2019
Patients, Family and Friends to Take Part in Variety of Rare Disease Day Events Worldwide

Scores of events are afoot worldwide to mark Feb. 28, Rare Disease Day 2019. The activities aim to raise awareness about rare diseases and the millions ... Read more
February 12, 2019February 12, 2019
ATS Releases Guidelines for Home Oxygen Therapy in Children with Sickle Cell Disease

The American Thoracic Society (ATS) released a set of new guidelines for home oxygen therapy for children with chronic health conditions that cause a ... Read more
February 7, 2019February 7, 2019
Emmaus Unveils Co-payment Assistance Program for SCD Endari Patients

For eligible sickle cell disease (SCD) patients covered by commercial insurance, biopharmaceutical company Emmaus Life Sciences will offer financial assistance to those who ... Read more
January 31, 2019January 31, 2019
FDA Revising ‘Draft Guidance’ on Developing Treatments for Rare Diseases

The U.S. Food and Drug Administration(FDA) is updating its 2015 draft guidelines for drug discovery in rare diseases, with new guidance on natural history— ... Read more
January 31, 2019January 31, 2019
Kids with Sickle Cell Anemia are More Sedentary than Healthy Peers, Study Suggests

Children and adolescents with sickle cell anemia do less physical activity and have a more sedentary lifestyle than healthy controls, according to a ... Read more

News

Registration Open for Sickle Cell Foundation of Georgia’s Annual Camp New Hope
January 29, 2019January 29, 2019
The Sickle Cell Foundation of Georgia (SCFG) will host its 42nd Annual Camp New ...
News

Vaso-Occlusive Pain Linked to Menstruation in Some Patients, Study Finds
January 24, 2019January 27, 2019
Acute vaso-occlusive pain is associated with the onset of menstruation, and about 25% of ...
News

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 ...
News

Howard University President Launches ‘Run To Cure Sickle Cell’ Campaign
January 18, 2019January 18, 2019
Howard University President Wayne A.I. Frederick has unveiled a campaign to raise awareness and funds for the ...
News

Randomized Trials Needed to Assess Benefits of Salmonella Vaccines in SCD Patients, Study Says
January 15, 2019January 15, 2019
Randomized controlled trials are needed to examine the benefits of vaccinating people with sickle ...
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 ...

Subscribe to our mailing list

Get regular updates delivered to your email box.

Thanks! Please check your email for a confirmation email.

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 ...
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 ...
Rising Healthcare Costs Strain EU Budgets Even as New Therapies Flourish
December 20, 2018December 20, 2018
LentiGlobin Shows Positive Effects in Severe Sickle Cell Disease Patients, Phase 1/2 Data Reports
December 18, 2018December 18, 2018
Voxelotor Can Promote Long-Term Benefits in Teens, Adults with SCD, Latest Clinical Data Show
December 11, 2018December 11, 2018
Delayed Hemolytic Transfusion Reaction Can Lead to Poor Outcome in SCD
December 4, 2018December 4, 2018

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 ...
New Tool Helps Primary Care Physicians Provide Better Care for SCD Patients, Study Shows
November 20, 2018November 20, 2018
School Staff Lacks Knowledge about Sickle Cell Disease, Student Survey Finds
November 13, 2018November 13, 2018
Phase 1 Trial to Test Under-the-Skin Injection of Sevuparin in Sickle Cell Patients
November 8, 2018November 8, 2018

Virtual Reality Program May Provide Effective Relief From Pain for SCD Patients, Study Suggests
November 1, 2018November 1, 2018
A virtual reality program designed to provide a distraction from pain may be an ...
#NORDSummit – Major Issues on Table for Rare Disease Patients in US as Midterm Elections Approach
October 30, 2018
Emmaus-sponsored ASCAT Conference Outlines Latest Sickle Cell Advances
October 30, 2018October 30, 2018

News

HemoType SC is a Fast, Inexpensive Diagnostic Tool for Sickle Cell Disease Screening, Study Shows
October 25, 2018October 25, 2018
The diagnostic test HemoType SC is a viable option for the highly sensitive, specific, inexpensive, ...
News

Marijuana Use Common in Sickle Cell Patients, Highlighting Need for More Research, Study Shows
October 23, 2018October 23, 2018
Many sickle cell disease (SCD) patients use marijuana for its medicinal benefits, according to an anonymous ...
News

Crizanlizumab Lowers Pain Crises in At-risk Sickle Cell Patients, Ad-hoc Trial Data Show
October 18, 2018October 18, 2018
Novartis’ investigational therapy for sickle cell disease (SCD), crizanlizumab (SEG101), was seen to reduce ...
News

Multi-Organization Partnership to Encourage Blood Donation and SCD Awareness
October 16, 2018October 16, 2018
A five-year partnership among The Sickle Cell Disease Association of America (SCDAA), the Sickle Cell ...
News

Agreement With Cardinal Health Widens Distribution of Endari for Emmaus
October 11, 2018October 11, 2018
A major distribution agreement with Cardinal Health will make Endari (L-glutamine) available in pharmacies nationwide ...
News

Advocacy Groups, Doctors Question Rising Prices of Rare Disease Treatments
October 9, 2018
Half a year has gone by since disgraced pharma executive Martin Shkreli was sentenced ...
News

#NORDSummit – More Than 700 Expected to Attend Oct. 15-16 Rare Disease Summit in Washington
September 21, 2018
More than 700 medical experts, pharmaceutical executives, patient advocates, and others are expected to ...
News

IP-10 Could Be Biomarker of Inflammation in Pregnant Sickle Cell Patients, Study Reports
September 20, 2018September 20, 2018
The blood level of a small protein called IP-10 could be a way to ...
News

Sickle Cell Patients in UK Survey, Especially Those 16 to 20, Voice Problems with Care and Pain Relief Given
September 18, 2018September 18, 2018
In a survey, patients across the U.K. with sickle cell disease — especially adolescents and young adults ...

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