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Pediatric Disease Articles & Analysis
38 news found
We believe that by using the same cells that promote tissue repair, organ maintenance, and immune system function, we can develop safe and effective therapies for some of the most difficult diseases and conditions. In 2024, we are focusing our efforts on two of our most promising programs: Hypoplastic Left Heart Syndrome (HLHS); and Alzheimer's Disease. HLHS: ...
Commercial and Pipeline Update Product portfolio Acute Care Hospital: RYANODEX®, vasopressin, Barhemsys®, Byfavo® Oncology: BENDEKA, BELRAPZO, PEMFEXY11, TREAKISYM Japan12 75-person commercial team covers all products excluding BENDEKA and TREAKISYM Company projects growth in earnings while still supporting R&D Cash flow from legacy products expected to continue to ...
ENA-001, a New Chemical Entity (NCE) with a novel mechanism of action as an agnostic respiratory stimulant, has previously been granted Rare Pediatric Disease designation for the Treatment of AoP by the FDA, with eligibility for a priority review voucher (PRV). ...
FIDELIO-DKD (FInerenone in reducing kiDnEy faiLure and dIsease prOgression in Diabetic Kidney Disease) investigated the efficacy and safety of finerenone in comparison to placebo in addition to standard of care on the reduction of kidney failure and kidney disease progression in approximately 5,700 patients with CKD and T2D. ...
ByBayer AG
FIDELIO-DKD (FInerenone in reducing kiDnEy faiLure and dIsease prOgression in Diabetic Kidney Disease) investigated the efficacy and safety of finerenone in comparison to placebo in addition to standard of care on the reduction of kidney failure and kidney disease progression in approximately 5,700 patients with CKD and T2D. ...
ByBayer AG
Oblato is currently developing OKN-007 as a treatment for glioblastoma multiforme (GBM). Pediatric diffuse gliomas are rare but have a very poor prognosis, and current treatment options show little benefit. ...
ByxCures
Persistent AoP can cause near- and long- term neurological development risks to the infant. ENA-001 has received Rare Pediatric Disease designation from the U.S. Food and Drug Administration (“FDA”) in the treatment of AoP, which potentially provides for a priority review voucher if the product is approved for this indication. ...
“We are proud soon to be able to offer physicians a new, disease-modifying intervention improving patients’ outcomes, by delaying their chronic kidney disease ...
ByBayer AG
Approximately half of patients with high-level BK viremia develop BKV nephropathy (BKVN), which can lead to decreased kidney survival and a return to end-stage renal disease and dialysis. There are no approved or effective antiviral treatments for BK viremia. ...
ByKalaris
About Leukocyte Adhesion Deficiency-I Severe Leukocyte Adhesion Deficiency-I (LAD-I) is a rare, autosomal recessive pediatric disease caused by mutations in the ITGB2 gene encoding for the beta-2 integrin component CD18. ...
“The multi-virus prevention approach is the most transformative use of posoleucel and, accordingly, we are seeing strong enthusiasm from hematologists and infectious disease specialists as we expand our Phase 3 study sites and ...
ByKalaris
(NASDAQ:LUMO), a clinical-stage biopharmaceutical company focused on therapeutics for rare diseases, initially targeting pediatric growth hormone deficiency, today announced a collaboration with Laura Dichtel, MD of Massachusetts General Hospital to evaluate the orally administered growth hormone (GH) secretagogue, LUM-201, in Nonalcoholic Fatty Liver ...
Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to its lead investigational multi-virus-specific T cell therapy, posoleucel, for the prevention of clinically significant infections and disease from six devastating viruses that commonly impact high-risk adult and pediatric patients following allogeneic ...
ByKalaris
About Endari® (prescription grade L-glutamine oral powder) Indication - Endari is indicated to reduce the acute complications of sickle cell disease in adult and pediatric patients five years of age and older. About Sickle Cell Disease There are approximately 100,000 people living with sickle cell disease (SCD) in the ...
About AlloVir AlloVir is a leading late clinical-stage cell therapy company with a focus on restoring natural immunity against life-threatening viral diseases in pediatric and adult patients with weakened immune systems. The company’s innovative and proprietary technology platforms leverage off-the-shelf, allogeneic, single- and multi-virus-specific T ...
ByKalaris
FIDELIO-DKD (FInerenone in reducing kiDnEy faiLure and dIsease prOgression in Diabetic Kidney Disease) investigated the efficacy and safety of finerenone in comparison to placebo in addition to standard of care on the reduction of kidney failure and kidney disease progression in approximately 5,700 patients with CKD and T2D. ...
ByBayer AG
The global, multi-center, randomized, double-blind, placebo-controlled study will enroll approximately 300 adult and pediatric patients and will evaluate the number of clinically significant infections or episodes of end-organ disease through the primary endpoint of the 14-week dosing interval. ...
ByKalaris
FIDELIO-DKD (FInerenone in reducing kiDnEy faiLure and dIsease prOgression in Diabetic Kidney Disease) investigated the efficacy and safety of finerenone in comparison to placebo in addition to standard of care on the reduction of kidney failure and kidney disease progression in approximately 5,700 patients with CKD and T2D. ...
ByBayer AG
About AlloVir AlloVir is a leading late clinical-stage cell therapy company with a focus on restoring natural immunity against life-threatening viral diseases in pediatric and adult patients with weakened immune systems. The company’s innovative and proprietary technology platforms leverage off-the-shelf, allogeneic, single- and multi-virus-specific T cells ...
ByKalaris
AdV-associated viral disease is among the leading causes of life-threatening complications following allo-HCT, often involving multiple vital organs, including the brain, lungs and liver, and occurs in 32% of pediatric and 6% of adult allo-HSCT patients. ...
ByKalaris