June 21, 2018June 21, 2018
Sickle Cell Disease Association Launches First Patient-powered Registry

The Sickle Cell Disease Association of America (SCDAA) recently launched the first patient-driven registry for sickle cell disease (SCD), ... Read more
June 19, 2018June 19, 2018
MaxCyte and NIH Office Partner to Develop Cell Therapy for Sickle Cell Disease

MaxCyte, a company specialized in cell-based medicines, announced it has entered in a research agreement with the National Heart, Lung ... Read more
June 13, 2018June 14, 2018
Potential Treatment Targeting Nitric Oxide Levels, Olinciguat, Named Orphan Drug by FDA

The U.S. Food and Drug Administration (FDA) recently granted orphan drug status to olinciguat (IW-1701) as a potential treatment of ... Read more
June 12, 2018June 12, 2018
Platform to Diagnose, Monitor SCD Wins Vodafone Wireless Innovation Project Grant

The Vodafone Americas Foundation recently announced the winners of its 10th annual Wireless Innovation Project (WIP), which awarded $600,000 in ... Read more
June 7, 2018June 7, 2018
Potential Sickle Cell Treatment Altemia Shows Promise in Phase 2 Trial, Data Reports

Sancilio Pharmaceuticals is reporting that its investigational oral compound Altemia (SC411, docosahexaenoic acid) showed an ability to safely treat sickle cell ... Read more
June 5, 2018June 5, 2018
FDA Places Clinical Hold on IND Application for CTX001 for Sickle Cell Disease

The U.S. Food and Drug Administration placed a clinical hold on the investigational new drug (IND) application for ... Read more
June 5, 2018June 5, 2018
US ‘Right to Try’ Law Meets with Mix of Praise and Criticism, Including Among Those with Rare Diseases

As President Trump signed the recently passed Right to Try legislation into law in a White House ceremony, Jordan ... Read more
May 31, 2018May 31, 2018
Loss of Cell Defense Mechanism Worsens Sickle Cell Disease Severity, Mouse Study Shows

Loss of an important part of the antioxidant cell defense system decreases production of hemoglobin in the blood and ... Read more
May 29, 2018May 29, 2018
Kids with Sickle Cell Disease Aren’t Receiving Key Vaccines, Michigan Study Finds

A significant proportion of children with sickle cell disease (SCD) are not receiving key recommended vaccines on time ... Read more
May 29, 2018May 29, 2018
#ERDC2018 – When Treating Rare Disease Patients, Don’t Overlook Quality of Life, Panel Urges

Finding treatments and potential cures for rare diseases is crucial, but so is the quality of patients’ lives — ... Read more
May 24, 2018May 24, 2018
Mutation Associated with Sickle Cell Disease Corrected in Patient’s Stem Cells, MaxCyte Reports

Global biotechnology company MaxCyte announced the successful correction of a genetic mutation associated with sickle cell disease (SCD) in ... Read more
May 22, 2018May 22, 2018
Sickle Cell Trait Not Associated with Increased Stroke Risk, Study Suggests

Sickle cell trait (SCT) is not associated with an increased incidence of stroke, a study in African-American patients suggests. ... Read more

June 21, 2018June 21, 2018

Sickle Cell Disease Association Launches First Patient-powered Registry

The Sickle Cell Disease Association of America (SCDAA) recently launched the first patient-driven registry for sickle cell disease (SCD), called Get Connected. The new registry was launched by the SCDAA on ... Read more

June 19, 2018June 19, 2018

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

MaxCyte, a company specialized in cell-based medicines, announced it has entered in a research agreement with the National Heart, Lung and Blood Institute (NHLBI) to develop potential therapies for sickle cell ... Read more

June 13, 2018June 14, 2018

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

The U.S. Food and Drug Administration (FDA) recently granted orphan drug status to olinciguat (IW-1701) as a potential treatment of sickle cell disease (SCD), Ironwood Pharmaceuticals announced. Evidence suggests that the ... Read more

June 12, 2018June 12, 2018

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

The Vodafone Americas Foundation recently announced the winners of its 10th annual Wireless Innovation Project (WIP), which awarded $600,000 in three-year grants to tackle critical issues facing the global community. ... Read more

June 7, 2018June 7, 2018

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

Sancilio Pharmaceuticals is reporting that its investigational oral compound Altemia (SC411, docosahexaenoic acid) showed an ability to safely treat sickle cell disease (SCD) in a Phase 2 study. Data from the Phase 2 ... Read more

June 5, 2018June 5, 2018

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

The U.S. Food and Drug Administration placed a clinical hold on the investigational new drug (IND) application for CTX001, an experimental therapy for the treatment of sickle cell disease, according ... Read more

June 5, 2018June 5, 2018

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

As President Trump signed the recently passed Right to Try legislation into law in a White House ceremony, Jordan McLinn of Indianapolis tried twice to embrace him. The third ... Read more

May 31, 2018May 31, 2018

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

Loss of an important part of the antioxidant cell defense system decreases production of hemoglobin in the blood and worsens spleen damage and inflammation in a mouse model of ... Read more

May 29, 2018May 29, 2018

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

A significant proportion of children with sickle cell disease (SCD) are not receiving key recommended vaccines on time or at all, according to a new study examining pneumococcal and ... Read more

May 29, 2018May 29, 2018

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

Finding treatments and potential cures for rare diseases is crucial, but so is the quality of patients’ lives — a rather nebulous term that means different things to different ... Read more

May 24, 2018May 24, 2018

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

Global biotechnology company MaxCyte announced the successful correction of a genetic mutation associated with sickle cell disease (SCD) in a patient’s hematopoietic stem cells (HSCs), the type of cells that ... Read more

May 22, 2018May 22, 2018

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

Sickle cell trait (SCT) is not associated with an increased incidence of stroke, a study in African-American patients suggests. The study, “Association of Sickle Cell Trait With Ischemic Stroke ... Read more