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

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

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

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

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

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

Study: Cannabidiol molecule contains more potent antioxidants

Study: Cannabidiol molecule contains more potent antioxidants

Study shows favorable impact of medication on cardiac structure and function

Study shows favorable impact of medication on cardiac structure and function

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

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

Immunotherapy combination shrinks rare angiosarcoma tumors in 25% of patients

Immunotherapy combination shrinks rare angiosarcoma tumors in 25% of patients

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

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

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

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

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

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

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

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

Researchers identify a promising method to stop COVID-19 infection

Researchers identify a promising method to stop COVID-19 infection

New, rare genetic form of dementia discovered

New, rare genetic form of dementia discovered

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

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

Gruthan Bioscience to develop new class of cholesterol-lowering drugs

Gruthan Bioscience to develop new class of cholesterol-lowering drugs

Providing personalized care for bleeding disorders

Providing personalized care for bleeding disorders

Cardiac MRI effective in identifying inflammation of the heart muscle in athletes

Cardiac MRI effective in identifying inflammation of the heart muscle in athletes

Oxytocinergic and vasopressinergic substances in the brain are involved in guiding social behavior

Oxytocinergic and vasopressinergic substances in the brain are involved in guiding social behavior

Advanced digital platform aims to improve outcomes for people with Angelman syndrome

Advanced digital platform aims to improve outcomes for people with Angelman syndrome

Triple therapy increases response rates in patients with Severe Aplastic Anemia

Triple therapy increases response rates in patients with Severe Aplastic Anemia