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Experts discuss the role of hormones in everyday life, highlight policy measures to address challenges

Experts discuss the role of hormones in everyday life, highlight policy measures to address challenges

A new 3D model for myotonic dystrophy

A new 3D model for myotonic dystrophy

New compound could alleviate neurological symptoms of CHARGE syndrome

New compound could alleviate neurological symptoms of CHARGE syndrome

CDISC, NORD announce partnership to develop global data standards for rare diseases

CDISC, NORD announce partnership to develop global data standards for rare diseases

C-Path opens access to D-RSC database to accelerate therapy development for DMD

C-Path opens access to D-RSC database to accelerate therapy development for DMD

Novel approach to gene editing bypasses disease-causing mutations in a gene

Novel approach to gene editing bypasses disease-causing mutations in a gene

New and more effective treatment for Pompe disease

New and more effective treatment for Pompe disease

Keele researcher awarded prestigious grant to identify treatments for devastating childhood disease

Keele researcher awarded prestigious grant to identify treatments for devastating childhood disease

Gift boosts efforts to understand and combat rare cancers

Gift boosts efforts to understand and combat rare cancers

New grant to Harvard Medical School is boosting efforts to understand and combat rare cancers

New grant to Harvard Medical School is boosting efforts to understand and combat rare cancers

Researchers evaluate new drug for spinal muscular atrophy in a broader spectrum of patients

Researchers evaluate new drug for spinal muscular atrophy in a broader spectrum of patients

Single injection of RNA therapy improves vision in patient with genetic form of childhood blindness

Single injection of RNA therapy improves vision in patient with genetic form of childhood blindness

Scientists discover new genetic disease that causes abnormal brain development in children

Scientists discover new genetic disease that causes abnormal brain development in children

Research consortium identifies cell stress mechanisms for chronic inflammatory liver disease

Research consortium identifies cell stress mechanisms for chronic inflammatory liver disease

Researchers develop first human model for studying the neuronal pathology of Leigh syndrome

Researchers develop first human model for studying the neuronal pathology of Leigh syndrome

Delays in TB diagnosis often exceed WHO’s recommendations, shows study

Delays in TB diagnosis often exceed WHO’s recommendations, shows study

Researcher uses alternative approach to find genetic drivers of rare eye disease

Researcher uses alternative approach to find genetic drivers of rare eye disease

Nutritional supplement may reduce risk of fatal strokes in patients with rare genetic disorder

Nutritional supplement may reduce risk of fatal strokes in patients with rare genetic disorder

Study: Early benefit assessment of new drugs within the framework of AM-NOG procedure

Study: Early benefit assessment of new drugs within the framework of AM-NOG procedure

UTHSC professor awarded $1.79 million grant to develop new amyloidosis treatment

UTHSC professor awarded $1.79 million grant to develop new amyloidosis treatment