Antibodies: The Key to Fighting Sickle Cell Anemia? Approval Could be Just Months Away
kalhh / Pixabay

Antibodies: The Key to Fighting Sickle Cell Anemia? Approval Could be Just Months Away

According to a story from PMLive, the US Food and Drug Administration (FDA) has recently begun the priority review process for a new potential therapy from pharmaceutical behemoth Novartis. This…

Continue Reading Antibodies: The Key to Fighting Sickle Cell Anemia? Approval Could be Just Months Away

Extremely Positive Results Announced from Interim Analysis of Phase 2 Clinical Trial for Hemochromatosis

Hereditary Hemochromatosis Hereditary hemochromatosis (HH) is a rare disease caused by hepcidin deficiency or hepcidin insensitivity. Hepcidin naturally regulates iron absorption/distribution in the body. Without hepcidin, HH patients suffer from…

Continue Reading Extremely Positive Results Announced from Interim Analysis of Phase 2 Clinical Trial for Hemochromatosis

Experimental Drug for Sickle Cell Disease Earns Fast Track Designation

According to a story from BioSpace, the biopharmaceutical company Imara, Inc. has recently announced that its experimental product candidate IMR-687 has earned Fast Track designation from the US Food and…

Continue Reading Experimental Drug for Sickle Cell Disease Earns Fast Track Designation

The Cure Rare Disease Foundation is Trying to Change the “One Size Fits All” Approach Toward Treatment

The Cure Rare Disease Foundation The Cure Rare Disease Foundation's mission is to increase collaborative efforts between researchers in order to accelerate the development of customized therapies for individuals living with…

Continue Reading The Cure Rare Disease Foundation is Trying to Change the “One Size Fits All” Approach Toward Treatment

Loma Linda University Cures Their First Sickle Cell Disease Patient with Stem Cell Transplant

LLUCH Loma Linda University Children's Hospital (LLUCH) is one of six hospitals in the University's health system. They are a faith-based system located in Southern California. They are the only…

Continue Reading Loma Linda University Cures Their First Sickle Cell Disease Patient with Stem Cell Transplant

Collaborative Effort to Improve Patient Access to Personalized Medicine

The FDA previously announced their plan to hire 50 new employees specifically to manage the influx of gene therapy applications. Currently, they have approximately 800 applications awaiting review and they…

Continue Reading Collaborative Effort to Improve Patient Access to Personalized Medicine

Positive Results from Phase 1/2 Study of Voxelotor as a Treatment for Sickle Cell Disease

Sickle cell disease (SCD) is a rare blood condition. It causes sickling in the red blood cells which results in the blockage of blood vessels and capillaries. The sickling process…

Continue Reading Positive Results from Phase 1/2 Study of Voxelotor as a Treatment for Sickle Cell Disease

Fast Track Designation Granted to Potential Therapy for Sickle Cell Disease

CRISPR Therapeutics and Vertex Pharmaceuticals have been collaborating to develop a new treatment option for Sickle Cell Disease. It's called CTX001, a type of stem cell therapy. This drug has…

Continue Reading Fast Track Designation Granted to Potential Therapy for Sickle Cell Disease
Positive Results from Part A of a Phase 3 Trial for Sickle Cell Disease
source: pixabay.com

Positive Results from Part A of a Phase 3 Trial for Sickle Cell Disease

Sickle cell disease (SCD) is a blood disorder. SCD patients have abnormal hemoglobin, or sickle hemoglobin, caused by a genetic mutation in the beta-chain. It results in deformed and inflexible…

Continue Reading Positive Results from Part A of a Phase 3 Trial for Sickle Cell Disease

Working to Improve Gene Therapy for Adrenoleukodystrophy and Other Rare Conditions

Rare disease research is often underrepresented in the field of science. However, recent developments have made researchers more interested in studying this group of diseases. For instance, investments in gene…

Continue Reading Working to Improve Gene Therapy for Adrenoleukodystrophy and Other Rare Conditions

A Gene Therapy for Sickle Cell Disease has Been Approved for an Investigational New Drug Application by the FDA

The United States Food and Drug Administration has accepted an Investigational New Drug application (IND) for an investigational gene therapy designed to be a treatment for sickle cell disease. Previously,…

Continue Reading A Gene Therapy for Sickle Cell Disease has Been Approved for an Investigational New Drug Application by the FDA
A Woman with Sickle Cell Disease Has Created a Foundation to Support Others with the Condition
allinonemovie / Pixabay

A Woman with Sickle Cell Disease Has Created a Foundation to Support Others with the Condition

The Noah’s Ark Foundation For Sickle Cell, an NGO based in Lagos, Nigeria, is working to support people with sickle cell disease. In a recent interview with the Nigerian Tribune,…

Continue Reading A Woman with Sickle Cell Disease Has Created a Foundation to Support Others with the Condition
Kids Fighting Cancer and Sickle Cell Disease are Showing Everyone Their Strength Through Photos
fotografierende / Pixabay

Kids Fighting Cancer and Sickle Cell Disease are Showing Everyone Their Strength Through Photos

September was Childhood Cancer Awareness Month and Sickle Cell Awareness Month. To mark to occasion, the Aflac Cancer and Blood Disorders Center at the Children’s Healthcare of Atlanta released a…

Continue Reading Kids Fighting Cancer and Sickle Cell Disease are Showing Everyone Their Strength Through Photos
Innovative Program in Greenville, SC Helps Patients With Sickle Cell Anemia
StockSnap / Pixabay

Innovative Program in Greenville, SC Helps Patients With Sickle Cell Anemia

According to a story from Greenville Online, the Greenville Health System has begun implementing a comprehensive treatment program for patients with sickle cell disease and its most severe and common…

Continue Reading Innovative Program in Greenville, SC Helps Patients With Sickle Cell Anemia
Is Fetal Gene Therapy the Cure to These Rare Diseases?
Free-Photos / Pixabay

Is Fetal Gene Therapy the Cure to These Rare Diseases?

Scientists are researching the possibility of fetal gene therapies as possible cures for rare diseases, such as Duchenne muscular dystrophy, hemophilia, sickle cell disease, and Gaucher's disease.  With fetal gene…

Continue Reading Is Fetal Gene Therapy the Cure to These Rare Diseases?
Researchers Investigated the Role of Technology in Self-Management of Sickle Cell Disease
kaboompics / Pixabay

Researchers Investigated the Role of Technology in Self-Management of Sickle Cell Disease

Researchers have investigated whether self-management electronic health (eHealth) interventions, such as text messaging, apps, and online cognitive-behavioural therapy, should be used to help patients with sickle cell disease self-manage the…

Continue Reading Researchers Investigated the Role of Technology in Self-Management of Sickle Cell Disease

ICYMI: Orphan Drug Designation Has Been Awarded to a Drug Being Developed to Treat Sickle Cell Disease

The experimental drug olinciguat (IW-1701) has been granted Orphan Drug Designation by the U.S. Food and Drug Administration (FDA) for the treatment of sickle cell disease. The full article can…

Continue Reading ICYMI: Orphan Drug Designation Has Been Awarded to a Drug Being Developed to Treat Sickle Cell Disease

Interim Data From a Study of a Sickle Cell Disease Gene Therapy Has Been Announced

The experimental gene therapy LentiGlobin for the treatment of severe sickle cell disease is being investigated in an ongoing Phase 1 clinical trial. Bluebird Bio, the company developing the therapy,…

Continue Reading Interim Data From a Study of a Sickle Cell Disease Gene Therapy Has Been Announced
Things to Know About Sickle Cell on World Sickle Cell Day
rawpixel / Pixabay

Things to Know About Sickle Cell on World Sickle Cell Day

Today is World Sickle Cell Day! Established in 2008, this international awareness day is aimed to increase public awareness and an understanding of sickle cell diseases, while educating those who may…

Continue Reading Things to Know About Sickle Cell on World Sickle Cell Day
The FDA has placed an Investigational Drug For Sickle Cell Disease on Clinical Hold
geralt / Pixabay

The FDA has placed an Investigational Drug For Sickle Cell Disease on Clinical Hold

The Investigational New Drug Application for the experimental drug CTX001 for treating sickle cell disease has been put on clinical hold, announced Vertex Pharmaceuticals Incorporated and CRISPR Therapeutics. This hold…

Continue Reading The FDA has placed an Investigational Drug For Sickle Cell Disease on Clinical Hold
Close Menu