Angelman syndrome
Sep 23, 2025
Merck Wins FDA Approval for KEYTRUDA QLEX for Subcutaneous Use in Adults With Solid Tumors; Incyte Gains FDA Nod for OPZELURA Cream in Children Aged 2–11 With Atopic Dermatitis; Minovia Therapeutics Receives FDA Fast Track Designation for MNV-201 in Myelodysplastic Syndrome; MavriX Bio Secures FDA Fast Track for MVX-220 Gene Therapy in Angelman Syndrome; Biocon Biologics Gets FDA Approval for Denosumab Biosimilars BOSAYA and AUKELSO
FDA Approves Merck’s KEYTRUDA QLEX for Subcutaneous Use Across Multiple Solid Tumors Merck announced that the FDA has approved KEYTRUDA QLEX (pembrolizumab and berahyaluronidase alfa-pmph) for subcutaneous administration in adults across most of KEYTRUDA’s approved solid tumor indications. Unlike the intravenous...
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Jul 01, 2025
Sobi’s GAMIFANT Approved by FDA as First Treatment for Macrophage Activation Syndrome in Still’s Disease; GSK Cuts RTS,S Malaria Vaccine Price for Children in Endemic Regions to Under $5; Schrödinger’s SGR-1505 Receives FDA Fast Track Designation for Relapsed/Refractory Waldenström Macroglobulinemia; Ultragenyx’s GTX-102 Granted FDA Breakthrough Therapy Designation for Angelman Syndrome; Eli Lilly’s AMYVID Label Updated by FDA to Support Alzheimer’s Disease Diagnosis
FDA Approves Sobi’s GAMIFANT as the First Treatment for Macrophage Activation Syndrome in Still’s Disease In a major advancement for rare inflammatory conditions, the FDA has approved GAMIFANT (emapalumab-lzsg) for the treatment of hemophagocytic lymphohistiocytosis (HLH)/macrophage activation syndrome (MA...
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Jan 18, 2021
Angelman Syndrome Market: Insights Into The Recent Late-Stage Drug Failures And Novel Approaches To Treating This Rare Neurogenetic Disorder
Angelman syndrome (AS) is a complex genetic rare disorder that affects the nervous system. The first sign of a child suffering from Angelman syndrome is a delay in development, such as the inability to sit without support or making incoherent babbling sounds. The hallmarks of the syndrome constitute severe physical...
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Jan 01, 2020
Angelman syndrome: A Rare Genetic Disorder
Angelman syndrome is a rare genetic disorder, which affects the nervous system. The people who are affected by the disease, show the characteristics like intellectual disability, severe speech impairment, delayed development and issues with movement and balance. Angelman syndrome causes are due to genetic abnor...
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Jan 09, 2019
Artificial Intelligence face-scanning app spots signs of rare genetic disorders
A range of rare genetic disorders is pinpointed by a deep-learning algorithm. This helps doctors and researchers by analysing pictures of people’s faces. Researchers describe the technology after the diagnostic aid, a smartphone app called Face2Gene. It relies on machine-learning algorithmsand brain-like neural...
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