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Weakness in lung and kidney cancer cells could serve as therapeutic target

Weakness in lung and kidney cancer cells could serve as therapeutic target

C-Path awarded FDA grant to establish Rare Disease Clinical Outcome Assessment Consortium

C-Path awarded FDA grant to establish Rare Disease Clinical Outcome Assessment Consortium

Recognizing CDKL5 Deficiency Disorder

Recognizing CDKL5 Deficiency Disorder

Two common Staph bacteria living on human skin exacerbate Netherton syndrome

Two common Staph bacteria living on human skin exacerbate Netherton syndrome

Gene therapy regenerates functional new neurons in mouse models of HD

Gene therapy regenerates functional new neurons in mouse models of HD

Study contributes to better understanding of rare KAT6A syndrome

Study contributes to better understanding of rare KAT6A syndrome

UCI neuro-oncologist uses comprehensive approach to battle brain cancer

UCI neuro-oncologist uses comprehensive approach to battle brain cancer

'Stranger Things' increased public awareness of cleidocranial dysplasia

'Stranger Things' increased public awareness of cleidocranial dysplasia

A143T variant of the GLA gene likely to cause late-onset Fabry cardiomyopathy

A143T variant of the GLA gene likely to cause late-onset Fabry cardiomyopathy

Fossilized dinosaur tail is home to a 60-million-year-old tumor

Fossilized dinosaur tail is home to a 60-million-year-old tumor

Finding the origins of angiosarcoma

Finding the origins of angiosarcoma

Study underscores the need for more precise rare disease definitions

Study underscores the need for more precise rare disease definitions

Study shows how gene therapy may help protect against vision loss in multiple sclerosis

Study shows how gene therapy may help protect against vision loss in multiple sclerosis

Research findings may one day help keep ALS at bay

Research findings may one day help keep ALS at bay

AAD honors board-certified dermatologists for treatment of a patient with aquagenic keratoderma

AAD honors board-certified dermatologists for treatment of a patient with aquagenic keratoderma

Better definition could lead to better diagnosis, treatment for rare diseases

Better definition could lead to better diagnosis, treatment for rare diseases

Research team publishes findings from study of TAF1 syndrome

Research team publishes findings from study of TAF1 syndrome

PCORI Board approves $70 million to support projects designed to improve care for health conditions

PCORI Board approves $70 million to support projects designed to improve care for health conditions

Tailored T-cells safe, may be effective in preventing and treating multiple viral infections

Tailored T-cells safe, may be effective in preventing and treating multiple viral infections

Newly identified antibody has potential to stop, reverse pulmonary arterial hypertension

Newly identified antibody has potential to stop, reverse pulmonary arterial hypertension