Sickle Cell Disease News and Research

Latest Sickle Cell Disease News and Research

FDA lifts partial clinical hold that paused enrollment of new patients in tazemetosta clinical trials

Epizyme, Inc., a clinical-stage company developing novel epigenetic therapies, today announced the U.S. Food and Drug Administration has lifted the partial clinical hold that had paused U.S.-based enrollment of new patients in its tazemetostat clinical trials.

24 Sep 2018

Study sheds light on how vaso-occlusive pain crises arise in sickle-cell disease

One of the most common complications of sickle-cell disease occurs when deformed red blood cells clump together, blocking tiny blood vessels and causing severe pain and swelling in the affected body parts.

4 Sep 2018

Study reveals molecular mechanisms underlying expansive arterial remodeling

Expansive arterial remodeling comprises a genetically programmed biological response designed to restore homeostatic levels of arterial wall stress after an increase in vessel flow load occurs.

25 Aug 2018

NIH grant for Mount Sinai to study use of inhaled corticosteroids for treatment of sickle cell disease

The Departments of Emergency Medicine and Hematology at the Icahn School of Medicine at Mount Sinai have been awarded a $4 million grant from the National Institutes of Health toward further study of inhaled corticosteroids to treat sickle cell disease in individuals who do not have asthma.

14 Aug 2018

Using CRISPR-Cas9 genome editing to correct mutation responsible for sickle cell disease

Genome editing using the CRISPR-Cas9 system has tremendous promise for therapeutic correction of genetic errors in human cells. Prior to adoption as a medical therapeutic, safety concerns relating to off-target effects must be minimized to mitigate risks that may arise from the unintended action of this system at sites similar, but not identical, to the desired gene target site.

From Integrated DNA Technologies 10 Aug 2018

Regenacy Pharmaceuticals partners with CMTA to advance ricolinostat for treating hereditary neuropathy

Regenacy Pharmaceuticals, LLC, a clinical-stage biopharmaceutical company developing breakthrough treatments for diabetic and other peripheral neuropathies, today announced a collaboration with the Charcot-Marie-Tooth Association, a registered non-profit organization serving the hereditary neuropathy patient community, to validate the role of HDAC6 in multiple forms of Charcot-Marie-Tooth disease and evaluate the efficacy of ricolinostat, a selective HDAC6 inhibitor, in animal models.

2 Aug 2018

Therapy with L-Glutamine reduces pain in patients with sickle cell disease

UCSF Benioff Children's Hospital Oakland clinical researchers, in conjunction with other sickle cell centers and scientists at Emmaus Life Sciences, Inc., have demonstrated that therapy with L-Glutamine reduced the frequency of pain episodes in both pediatric and adult patients with sickle cell disease.

27 Jul 2018

Researchers identify enzyme as potential new drug target for blood disorders

Medical researchers have identified a key signaling protein that regulates hemoglobin production in red blood cells, offering a possible target for a future innovative drug to treat sickle cell disease.

20 Jul 2018

CRISPR/Cas9 gene editing can cause greater genetic damage than previously thought

Scientists at the Wellcome Sanger Institute have discovered that CRISPR/Cas9 gene editing can cause greater genetic damage in cells than was previously thought. These results create safety implications for gene therapies using CRISPR/Cas9 in the future as the unexpected damage could lead to dangerous changes in some cells.

16 Jul 2018

New guideline recommends first-trimester screening of pregnant women for asymptomatic bacteriuria

First-trimester screening of pregnant women for asymptomatic bacteriuria -- higher than normal bacteria levels without symptoms of a bladder infection -- is recommended by the Canadian Task Force on Preventive Health Care in an updated guideline in CMAJ

9 Jul 2018

Music therapy shows promise for improving lives of people with sickle cell disease

A new study published in the Journal of Music Therapy reveals promising findings for the application of improvisational music therapy in assuaging the multidimensional acute pain of adult patients with sickle cell disease.

14 Jun 2018

Acetaminophen use helps reduce acute kidney injury risk in pediatric heart surgery patients

Children who underwent cardiac surgery were less likely to develop acute kidney injury if they had been treated with acetaminophen in the first 48 hours after their procedures, according to a Vanderbilt study just published in JAMA Pediatrics.

15 May 2018

Study: Stigmatizing language in medical records may play a role in how the patient is treated

A Johns Hopkins study found that physicians who use stigmatizing language in their patients' medical records may affect the care those patients get for years to come.

9 May 2018

Doctors cure sickle cell patients using allogeneic stem cells

Seven adults with sickle cell disease have been cured by doctors at the University of Illinois Hospital, thanks to the development of a new protocol for stem cell transplantation.

25 Apr 2018

Pitt researcher secures nearly $5 million NIH funding for 'Tic-Tac-Toe' RF coil system

The University of Pittsburgh houses a whole-body 7 Tesla magnetic resonance imager (7T MRI), one of the strongest human MRI devices in the world and a powerful imaging tool that allows researchers to gain a far better understanding of brain structure and function.

13 Apr 2018

Using Cord Blood to Alleviate the Symptoms of Autism

Cord blood has been studied extensively for the treatment of hematological diseases such as cancer and sickle cell anemia, but is now in phase II trials for the alleviation of autism. Dr. Ann Smith, director of Smart Cells who provided the cord blood for the trial, discusses the research and it's potential.

9 Apr 2018

Genetic mutation that may protect people from malaria found to be more common

A genetic mutation that may protect people from malaria, but was thought to be rare, is surprisingly common, suggest the findings of a new study led by scientists at The Scripps Research Institute.

23 Mar 2018

University of Illinois at Chicago awarded $4.6 million NIH grant for chronic pain research

The University of Illinois at Chicago has been awarded a $4.6 million grant from the National Heart, Lung, and Blood Institute to study the molecular neurobiology of chronic pain in patients with sickle cell disease and to develop potential new drug treatments.

26 Feb 2018

Study reveals HCT as effective treatment for NHL patients regardless of age

Results from a retrospective study of 1,629 patients with non-Hodgkin lymphoma showed that survival at 4 years following allogeneic hematopoietic cell transplantation for patients age 65 years and older is comparable to patients age 55 to 64 years.

21 Feb 2018

Combination chemotherapy increases survival in animal models of glioblastoma

A diagnosis of the brain cancer glioblastoma carries a dismal prognosis, with most patients dying within five years.

23 Jan 2018

Sickle Cell Disease News and Research

Latest Sickle Cell Disease News and Research

FDA lifts partial clinical hold that paused enrollment of new patients in tazemetosta clinical trials

Study sheds light on how vaso-occlusive pain crises arise in sickle-cell disease

Study reveals molecular mechanisms underlying expansive arterial remodeling

NIH grant for Mount Sinai to study use of inhaled corticosteroids for treatment of sickle cell disease

Using CRISPR-Cas9 genome editing to correct mutation responsible for sickle cell disease

Regenacy Pharmaceuticals partners with CMTA to advance ricolinostat for treating hereditary neuropathy

Therapy with L-Glutamine reduces pain in patients with sickle cell disease

Researchers identify enzyme as potential new drug target for blood disorders

CRISPR/Cas9 gene editing can cause greater genetic damage than previously thought

New guideline recommends first-trimester screening of pregnant women for asymptomatic bacteriuria

Music therapy shows promise for improving lives of people with sickle cell disease

Acetaminophen use helps reduce acute kidney injury risk in pediatric heart surgery patients

Study: Stigmatizing language in medical records may play a role in how the patient is treated

Doctors cure sickle cell patients using allogeneic stem cells

Pitt researcher secures nearly $5 million NIH funding for 'Tic-Tac-Toe' RF coil system

Using Cord Blood to Alleviate the Symptoms of Autism

Genetic mutation that may protect people from malaria found to be more common

University of Illinois at Chicago awarded $4.6 million NIH grant for chronic pain research

Study reveals HCT as effective treatment for NHL patients regardless of age

Combination chemotherapy increases survival in animal models of glioblastoma

Achieving and maintaining ISO/IEC 27001 and BS 10012 certifications

Expanding research and clinical options for children with cancer

Examining how pain could play a direct protective role in the gut

Sickle Cell Disease News and Research

Latest Sickle Cell Disease News and Research

Achieving and maintaining ISO/IEC 27001 and BS 10012 certifications

Expanding research and clinical options for children with cancer

Examining how pain could play a direct protective role in the gut

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