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Newly approved gene therapy for SMA is based on delivery vehicle discovered by Penn expert

Newly approved gene therapy for SMA is based on delivery vehicle discovered by Penn expert

Largest study of women with hyperemesis gravidarum launches in Europe

Largest study of women with hyperemesis gravidarum launches in Europe

Bioactive compounds in cocoa bean extract could be used to develop new treatment for skeletal diseases

Bioactive compounds in cocoa bean extract could be used to develop new treatment for skeletal diseases

CU Anschutz researchers identify new immunodeficiency disease

CU Anschutz researchers identify new immunodeficiency disease

Scientists discover first gene involved in lower urinary tract obstruction

Scientists discover first gene involved in lower urinary tract obstruction

Summer bummer: A young camper’s $142,938 snakebite

Summer bummer: A young camper’s $142,938 snakebite

Pregnant women with type 1 diabetes are at greater risk of preterm birth

Pregnant women with type 1 diabetes are at greater risk of preterm birth

Study provides new insights into regulatory T cells' role in protecting against autoimmune disease

Study provides new insights into regulatory T cells' role in protecting against autoimmune disease

Researchers identify cause of inherited metabolic disorder

Researchers identify cause of inherited metabolic disorder

Study demonstrates viability of gene editing strategy for recessive dystrophic epidermolysis bullosa

Study demonstrates viability of gene editing strategy for recessive dystrophic epidermolysis bullosa

Inherited form of rickets improves more with new injectable medicine than conventional therapy

Inherited form of rickets improves more with new injectable medicine than conventional therapy

Royal Holloway professors to lead new to research into curing Neurofibromatosis type 1

Royal Holloway professors to lead new to research into curing Neurofibromatosis type 1

Fear center in the brain provides protection against body perception disorders

Fear center in the brain provides protection against body perception disorders

Researchers unravel how the immune system responds to diffuse cutaneous leishmanisis

Researchers unravel how the immune system responds to diffuse cutaneous leishmanisis

Researchers find cause, treatment for CVID lung disease using precision medicine

Researchers find cause, treatment for CVID lung disease using precision medicine

Liver tissue model can help investigate the effects of RNA interference

Liver tissue model can help investigate the effects of RNA interference

Causes of disease including infant cancer can be revealed with new tool from Princeton University research team

Causes of disease including infant cancer can be revealed with new tool from Princeton University research team

New multi-year affiliation announced to establish global center for rare diseases

New multi-year affiliation announced to establish global center for rare diseases

Drug increases melanin production in some people with albinism

Drug increases melanin production in some people with albinism

BGU develops new AI platform for monitoring and predicting ALS progression

BGU develops new AI platform for monitoring and predicting ALS progression