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Researchers establish BRIDGES program to synergize diagnostic efforts, patient outcome management

For over 20 years, the Genetics Service at the KK Women's and Children's Hospital (KKH) has been providing clinical care to patients with genetic disorders.

22 Dec 2020

Neanderthal gene variant increases risk of severe COVID-19

Researchers in Germany have conducted a study showing that between 2 and 8% of people in Eurasia carry a genetic variant inherited from Neanderthals that significantly increases the risk of becoming critically ill with coronavirus disease 2019 (COVID-19).

15 Dec 2020

Novel antibody-drug conjugate shows 29% response rate in rare blood cancer

Early study data shows a 29% overall response rate, the favorable safety profile in blastic plasmacytoid dendritic cell neoplasm (BPDCN)

8 Dec 2020

Clinical investigators present abstracts on hematological malignancies at ASH Annual Meeting and Exposition

Clinical investigators from Hackensack Meridian Health John Theurer Cancer Center (JTCC), a member of the Georgetown Lombardi Comprehensive Cancer Center consortium, are to present updates on treatment advances in multiple myeloma (MM), mantle cell lymphoma (MCL), and other types of B-cell lymphoma (BCL) as well as leukemia at the 62nd American Society of Hematology (ASH) Annual Meeting and Exposition, to be held virtually from December 5-8, 2020.

7 Dec 2020

Genethon to start international clinical trial for treatment of Duchenne muscular dystrophy

Genethon, dedicated to designing and developing gene therapy products for rare diseases, received this Monday 30th of November the authorization from the ANSM, the French National Agency for Medicines and Health Products Safety, to start in France a multicentre international clinical trial for the treatment of Duchenne muscular dystrophy with product GNT 004.

2 Dec 2020

University of Sheffield and Pfizer launch new consortium to accelerate development of ATMPs

The University of Sheffield and Pfizer have today (23 November 2020) launched a new consortium which aims to standardize and accelerate the development of Advanced Therapy Medicinal Products (ATMPs), allowing potentially transformative treatments to reach patients sooner.

23 Nov 2020

Immunotherapies effective against lung cancer, melanoma may also work against rare skin tumor

Cutaneous angiosarcoma is a rare, highly aggressive skin tumor found primarily on the scalps of older White people, but based on new research from the University of North Carolina School of Medicine and the Lineberger Comprehensive Cancer Center, immunotherapies effective against lung cancer and melanoma may also work against this cancer.

18 Nov 2020

Study: Cannabidiol molecule contains more potent antioxidants

Nowadays, cannabidiol is a star component, not only in the world of cosmetics, but also in pharmaceutics and nutrition due to its antioxidant properties and its therapeutical potential.

17 Nov 2020

Study shows favorable impact of medication on cardiac structure and function

For the first time, medication has impacted heart muscle thickness and function for patients with the most common inherited heart condition, rather than simply addressing their symptoms.

17 Nov 2020

First-ever phase 3 advanced anal cancer trial opens for patient enrollment

The first randomized phase 3 clinical trial ever to be conducted in patients with advanced cancer of the anal canal is now open and enrolling patients.

16 Nov 2020

Immunotherapy combination shrinks rare angiosarcoma tumors in 25% of patients

Researchers from SWOG Cancer Research Network, a cancer clinical trials group funded by the National Cancer Institute's Division of Cancer Diagnosis and Treatment, part of the National Institutes of Health, have shown that the immunotherapy combination of ipilimumab and nivolumab shrinks rare angiosarcoma tumors in 25 percent of all patients, with some having an even stronger response to the drug combination.

15 Nov 2020

Researchers receive federal grant to develop tool that protects privacy in brain imaging analyses

Georgia State University faculty have received a five-year, $3 million federal grant to further develop a tool that will allow researchers around the world to participate in extensive brain imaging analysis without sharing protected patient data.

10 Nov 2020

Trend analysis in Japan reveals Creutzfeldt-Jakob disease may not be so rare anymore

Creutzfeldt-Jakob disease (CJD) is a debilitating disorder that causes rapid degeneration of the brain as well as progressing dementia.

19 Oct 2020

Two studies show the benefit of collaborative research on childhood blood disorders

When a child has a rare blood disorder, clinicians can struggle to find the best diagnostic and treatment methods.

15 Oct 2020

FDA awards six clinical trial research grants to enhance development of therapies for rare diseases

Today, the U.S. Food and Drug Administration announced that it has awarded six new clinical trial research grants to principal investigators from academia and industry totaling over $16 million over the next four years.

8 Oct 2020

Researchers identify a promising method to stop COVID-19 infection

While the world waits eagerly for a safe and effective vaccine to prevent infections from severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2), the virus behind the COVID-19 pandemic, researchers also are focusing on better understanding how SARS-CoV-2 attacks the body in the search for other means of stopping its devastating impact.

8 Oct 2020

New, rare genetic form of dementia discovered

A new, rare genetic form of dementia has been discovered by a team of Penn Medicine researchers. This discovery also sheds light on a new pathway that leads to protein build up in the brain -- which causes this newly discovered disease, as well as related neurodegenerative diseases like Alzheimer's Disease -- that could be targeted for new therapies.

1 Oct 2020

Cincinnati Children's research leads to approval of new drug for hypereosinophilic syndrome

Following two decades of research on a group of rare diseases called hypereosinophilic syndrome at Cincinnati Children's Hospital Medical Center, the U.S. Food and Drug Administration has approved the drug Nucala (mepolizumab) for use in the treatment of patients with hypereosinophilic syndrome.

28 Sep 2020

Gruthan Bioscience to develop new class of cholesterol-lowering drugs

Gruthan Bioscience, LLC, a Medical University of South Carolina startup based in Charleston, South Carolina, received a Small Business technology Transfer (STTR) award from the National Heart, Blood and Lung Institute in August to take the next step in developing a novel class of cholesterol-lowering drugs to treat familial hypercholesterolemia.

24 Sep 2020

Providing personalized care for bleeding disorders

An interview with Dr. Carmen Escuriola-Ettingshausen MD, Mörfelden-Walldorf, Germany on providing personalized care for bleeding disorders.

From Takeda 21 Sep 2020