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Leadiant Biosciences announces FDA approval of Revcovi for treating pediatric, adult ADA-SCID patients

Leadiant Biosciences announces FDA approval of Revcovi for treating pediatric, adult ADA-SCID patients

Alnylam announces positive results from Phase 1/2 study of lumasiran for treating primary hyperoxaluria-1

Alnylam announces positive results from Phase 1/2 study of lumasiran for treating primary hyperoxaluria-1

Alnylam submits CTA application to MHRA to initiate Phase 1/2 study of ALN-AAT02

Alnylam submits CTA application to MHRA to initiate Phase 1/2 study of ALN-AAT02

New project identifies immune checkpoint inhibition as potential treatment for angiosarcomas

New project identifies immune checkpoint inhibition as potential treatment for angiosarcomas

Lumos acquires license for LUM-201 drug that promotes secretion of growth hormone

Lumos acquires license for LUM-201 drug that promotes secretion of growth hormone

Alexion announces positive results from Phase 3 PREVENT study of Soliris in patients with NMOSD

Alexion announces positive results from Phase 3 PREVENT study of Soliris in patients with NMOSD

Salk research in yeast leads to serendipitous finding about hypomyelinating leukodystrophy

Salk research in yeast leads to serendipitous finding about hypomyelinating leukodystrophy

Investigators identify group of blood metabolites that could help detect autism

Investigators identify group of blood metabolites that could help detect autism

BeiGene collaborates with SpringWorks to evaluate new combinational therapy for solid tumors

BeiGene collaborates with SpringWorks to evaluate new combinational therapy for solid tumors

Pfizer terminates clinical studies evaluating domagrozumab for treatment of Duchenne muscular dystrophy

Pfizer terminates clinical studies evaluating domagrozumab for treatment of Duchenne muscular dystrophy

Eagle Pharmaceuticals completes enrollment of second clinical study to evaluate safety, efficacy of RYANODEX

Eagle Pharmaceuticals completes enrollment of second clinical study to evaluate safety, efficacy of RYANODEX

FDA approves first drug for treatment of rare disease affecting the cornea

FDA approves first drug for treatment of rare disease affecting the cornea

RDMD raises $3 million in seed funding to accelerate rare disease research, drug development

RDMD raises $3 million in seed funding to accelerate rare disease research, drug development

Insight into endocrine cancers and treatment options

Insight into endocrine cancers and treatment options

Scientists pinpoint the cause of a rare childhood seizure disorder

Scientists pinpoint the cause of a rare childhood seizure disorder

Researchers discover genetic mutation underlying debilitating childhood brain disorder

Researchers discover genetic mutation underlying debilitating childhood brain disorder

EMA grants orphan designation to selumetinib for treatment of neurofibromatosis type 1

EMA grants orphan designation to selumetinib for treatment of neurofibromatosis type 1

CHOP experts identify new autoimmune disorder along with personalized treatment for young patient

CHOP experts identify new autoimmune disorder along with personalized treatment for young patient

Regenacy Pharmaceuticals partners with CMTA to advance ricolinostat for treating hereditary neuropathy

Regenacy Pharmaceuticals partners with CMTA to advance ricolinostat for treating hereditary neuropathy

Researchers reveal molecular mechanisms that make people with EV prone to ß-HPVs

Researchers reveal molecular mechanisms that make people with EV prone to ß-HPVs