Pediatric Disease Articles & Analysis
21 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). ...
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. ...
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. ...
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 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
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
The Company is planning to initiate additional clinical studies for ENA-001 across multiple indications in the coming year, including for Apnea of Prematurity for which it has received the designation of Rare Pediatric Disease by 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
Enalare is planning to develop this unique agnostic respiratory stimulant for a multitude of indications related to acute respiratory depression, including for neonates experiencing Apnea of Prematurity. Under the FDA's rare pediatric disease designation program, the FDA may grant a priority review voucher to a sponsor who receives a product approval for a rare ...
This is the largest prospective study in pediatrics for our technology to date, and part of a growing evidence base generated by multiple clinical groups over the past decade, establishing the high performance and utility of our technology. ...
ByMeMed
For children and adolescents with chronic kidney disease (CKD), as for adults, the unmet need is high for new treatments to delay disease progression and preserve kidney function / The Phase III study FIONA will investigate the effect of finerenone in pediatric patients with CKD and severely increased proteinuria Bayer announced today the ...
ByBayer AG
The FDA has granted HemoShear’s HST5040 Orphan Drug, Fast Track and Rare Pediatric Disease designations to treat MMA and PA. “There are currently no targeted pharmacologic treatments for MMA or PA that can improve quality of life or extend lifespan for these devastating diseases,” said Kimberly Chapman, MD, PhD, genetic ...
” About HST5040 The FDA has granted HemoShear’s HST5040 Orphan Drug, Fast Track and Rare Pediatric Disease designations for the treatment of MMA and PA. HemoShear’s HERO phase 2 clinical study of HST5040 will enroll at least 12 patients aged 2 and older with MMA or PA at select children’s hospitals in the United States. ...
The Company is developing a pipeline-in-a-product opportunity with Tadekinig alfa in a wide range of IL-18 mediated diseases where hyperinflammation or ‘cytokine storm’, is an issue, including COVID-19. Tadekinig alfa is currently in late-stage development for the treatment of severe orphan autoinflammatory diseases, including primary and secondary ...
Broncho-Vaxom® is a therapeutic agent that effectively prevents and treats recurrent or acute respiratory infections in adult and pediatric patients by boosting the host immunity. In China, recurrent respiratory tract infection is a common disease in pediatrics, with an incidence rate of about 20% 1. ...
"Antibiotics are frequently used to prevent disease when food animals are kept in large scale unsanitary factory farming conditions. ...
The U.S. FDA defines a “rare pediatric disease” as a serious or life-threatening disease primarily affecting individuals age 18 years or younger that impacts fewer than 200,000 individuals in the United States. The Rare Pediatric Disease designation provides incentives to advance the development of ...