Rare Disease News and Research

RSS
New taxonomic classification of non-skeletal rare congenital disorders with impact on bone physiology

New taxonomic classification of non-skeletal rare congenital disorders with impact on bone physiology

Study: TTR protein plays a protective role in Alzheimer's disease

Study: TTR protein plays a protective role in Alzheimer's disease

RedHill Biopharma: RHB-204 receives FDA Fast Track designation for treatment of NTM disease

RedHill Biopharma: RHB-204 receives FDA Fast Track designation for treatment of NTM disease

Trinity team uncovers mutations linked with early onset dementia

Trinity team uncovers mutations linked with early onset dementia

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

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

Neanderthal gene variant increases risk of severe COVID-19

Neanderthal gene variant increases risk of severe COVID-19

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

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

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

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

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