Legacy MEDSearch

JUNE 28, 2023

1 Early treatment of PWS during childhood leads to the best clinical response. 1 Early treatment of PWS during childhood leads to the best clinical response. The Vbeam 595 nm PDL is world recognized as the gold standard treatment for PWS 4,5 and the primary choice for hemangioma treatment in pediatric patients. Geronemus, M.D.,

Medical 52

Medical FDA Recruitment Side effects 52

Pharmaceutical Technology

SEPTEMBER 22, 2022

Avrobio has received rare pediatric disease designation from the US Food and Drug Administration (FDA) for its investigational gene therapy, AVR-RD-04, designed to treat cystinosis. The FDA's rare pediatric disease designation and Voucher Program aid the development of new drugs and biologics to prevent and treat rare paediatric ailments.

Food and Drug Administration 59

Food and Drug Administration FDA Food Pharmaceutical 59

Medgadget

NOVEMBER 15, 2022

SpineX , a medtech company based in California, has developed the Spinal Cord Innovation in Pediatrics (SCiP) device, a non-invasive spinal cord neuromodulation technology that is intended to treat children with cerebral palsy. The children received two interventions a week during the study.

Patients 97

Patients 97

European Pharmaceutical Review

MARCH 4, 2024

Importance of the new data for the Novartis gene therapy This data is “the first Zolgensma open-label clinical study to include older and heavier, as well as previously treated patients,” according to Dr Sandra Reyna, Chief Scientific Advisor and Head of Global Medical Engagement for SMA at Novartis.

Patients 81

Patients Safety Medical Pharmaceutical 81

PM360

FEBRUARY 12, 2024

More than 70% of rare diseases are genetic in nature with nearly half of them impacting pediatric patients. Since rates in children are generally low, every pediatric cancer is deemed rare. Nearly 80% of clinical trials fail to finish on schedule with 20% delayed over six months or more.

Food and Drug Administration 105

Food and Drug Administration Patients Networking Transportation 105

Pharmacy Times

JUNE 16, 2023

Imposing the FDA on-label/off-label framework on administratively defined "children" resulted in a regulatory demand for pediatric studies that had no basis in clinical medicine, with the exception of the small group of preterm newborns.

Medicine 52

Medicine FDA 52

Pharmacy Times

MAY 31, 2023

Pediatric drug development, which has a complex background and history, has fundamental catches based around how children are defined in the context of clinical studies.

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Legacy MEDSearch

MAY 10, 2023

The ANNE® One platform is now cleared as a clinical-grade continuous monitoring solution for neonates, infants, and adults. “We see the ANNE® One system to be broadly applicable across the entire clinical care continuum from pediatrics to adults,” added JooHee Lee, Senior Product Manager and cofounder of Sibel Health.

FDA 52

FDA Food and Drug Administration Recruitment Medical 52

Pharmaceutical Technology

MAY 17, 2023

TAK-755 is the first and only treatment in clinical development that provides targeted replacement of ADAMTS13, addressing the underlying cause of the disease. “We The Phase III trial assessed TAK-755’s clinical benefit across several clinically relevant endpoints, against plasma-based therapies, in a randomised cross-over study.

Food and Drug Administration 97

Food and Drug Administration FDA Food Patients 97

European Pharmaceutical Review

APRIL 3, 2023

These results have led to a major amendment in a European Phase III paediatric clinical trial. The oncology treatment showed clinical activity across patients of all ages with the three neuroblastoma-specific hotspot ALK mutations. The data showed 63 percent of patients responded to the combined treatment.

Food and Drug Administration 78

Food and Drug Administration Medicine Patients Food 78

Pharmaceutical Technology

JUNE 5, 2023

Kid EDV is under clinical development by ImmunityBio and currently in Phase I for Pediatric Diffuse Intrinsic Pontine Glioma. According to GlobalData, Phase I drugs for Pediatric Diffuse Intrinsic Pontine Glioma does not have sufficient historical data to build an indication benchmark PTSR for Phase I.

Leads 52

Leads 52

Pharmaceutical Technology

JANUARY 17, 2023

Orphagen Pharmaceuticals has received a rare pediatric disease designation (RPDD) for OR-449 from the US Food and Drug Administration (FDA) to treat paediatric adrenocortical carcinoma (ACC). The post Orphagen’s ACC therapy receives FDA rare pediatric disease status appeared first on Pharmaceutical Technology.

Food and Drug Administration 59

Food and Drug Administration FDA Food Pharmaceutical 59

European Pharmaceutical Review

AUGUST 31, 2023

Based on new data from the Novartis Phase I/II clinical trial , which used the first therapy to target a new genetic area and use cryopreserved stem cells, trial participants reported a decrease in vaso-occlusive events. This is where sickled red blood cells accumulate and cause a blockage.

Food and Drug Administration 110

Food and Drug Administration Medicine Food FDA 110

pharmaphorum

NOVEMBER 11, 2022

Leading with practical insights from the best in the industry, the 4th Annual Inflammasome Therapeutics Summit is returning in person on November 29 – December 1 to Boston to equip you with the knowledge and connections to propel the next frontier of inflammasome therapeutics from pipeline to clinical and commercial reality.

Medicine 52

Medicine Medical Leads Pharma 52

European Pharmaceutical Review

SEPTEMBER 8, 2022

Lurie Children’s Hospital of Chicago and Professor of Pediatrics at Northwestern University Feinberg School of Medicine, both US. Regulators call for clinical research to include pregnant and breastfeeding women… The post Dolutegravir shows promise in pregnancy appeared first on European Pharmaceutical Review.

Medicine 99

Medicine Safety Physicians Pharmaceutical 99

Pharmaceutical Technology

FEBRUARY 21, 2023

The findings showed that 100% of patients had quick and sustained normalisation of serum albumin (a disease biomarker) and improvement or no worsening of clinical symptoms at 24 weeks. It was also granted Orphan Drug Designation during the same time.

Food and Drug Administration 52

Food and Drug Administration FDA Food Safety 52

Pharma Leaders

APRIL 14, 2023

The gene therapy candidate is being evaluated in a phase 1/2 clinical trial, in which six patients ages four to 11 years are expected to enroll for a one-time intravenous dose of the medication. The agency previously granted RGX-202 Orphan Drug and Rare Pediatric Disease designations. Related Topics

FDA 52

FDA Medical Patients 52

European Pharmaceutical Review

JULY 25, 2023

Additionally, in the 0.3mg/kg group, 55 percent of patients experienced a clinically meaningful improvement in symptoms. more effective treatment options for this significant complication are desperately needed,” urged Dr Carrie Kitko, Medical Director of the Pediatric Stem Cell Transplant Program at the Vanderbilt-Ingram Cancer Center.

Food and Drug Administration 97

Food and Drug Administration FDA Food Medical 97

pharmaphorum

JULY 5, 2022

With more than 500 dermatology clinical trials currently underway, it is often heard that there is a ‘’competition for participants’’. Instead, atopic dermatitis clinical trial organisers often simply only reach out to a limited patient population and fail to reach the complete, diverse, patient population of atopic dermatitis.

Recruitment 95

Recruitment Competition Patients Marketing 95

Pharmaceutical Technology

JUNE 5, 2023

Pralsetinib is under clinical development by F. Attributes of the drug, company and its clinical trials play a fundamental role in drug-specific PTSR and likelihood of approval. Hoffmann-La Roche and currently in Phase II for Pancreatic Cancer. Pralsetinib overview Pralsetinib (Gavreto) is an anticancer agent.

Patients 40

Patients Medicine Healthcare Healthcare 40

Pharmaceutical Technology

FEBRUARY 9, 2023

Ibutamoren mesylate is under clinical development by Lumos Pharma and currently in Phase I for Turner Syndrome. Attributes of the drug, company and its clinical trials play a fundamental role in drug-specific PTSR and likelihood of approval.

Pharma 40

Pharma Leads Patients Medical 40

Pharmaceutical Technology

OCTOBER 6, 2022

Additionally, GBT’s pipeline of preclinical and clinical investigational assets for SCD includes GBT021601 (GBT601) and inclaclumab. The FDA has granted Orphan Drug and Rare Pediatric Disease designations for these two assets.

Food and Drug Administration 115

Food and Drug Administration FDA Food Pharmaceutical 115

Pharmacy Times

AUGUST 17, 2022

Zynteglo was approved for the treatment of adult and pediatric patients with beta-thalassemia who require regular red blood cell transfusions after safety and efficacy were demonstrated in clinical trials.

Patients 49

Patients Safety FDA 49

Pharmaceutical Technology

FEBRUARY 9, 2023

Ibutamoren mesylate is under clinical development by Lumos Pharma and currently in Phase II for Growth Hormone Deficiency. Attributes of the drug, company and its clinical trials play a fundamental role in drug-specific PTSR and likelihood of approval.

Pharma 40

Pharma Leads Patients Medical 40

European Pharmaceutical Review

FEBRUARY 8, 2024

Vertex Pharmaceuticals’ once-daily small molecule vanzacaftor/tezacaftor/deutivacaftor (vanza triple) for cystic fibrosis (CF) has gleaned positive results in Phase III trials.

Food and Drug Administration 91

Food and Drug Administration Medicine Medical Consulting 91

Pharmaceutical Technology

SEPTEMBER 16, 2022

Currently in the clinical stage, the antibody is being developed as a stem cell transplant conditioning agent. In an ongoing multicentre clinical trial, JSP191 has so far been analysed in 14 patients with SCID. It aids in creating a vacant space for the donor or gene-corrected transplanted stem cells to be engrafted. .

Food and Drug Administration 52

Food and Drug Administration FDA Food Patients 52

Legacy MEDSearch

JULY 11, 2023

The functionality and performance of the Neoasis ® were highlighted in a recent presentation at the Pediatric Academic Society conference. “In In previously published clinical studies, this level of noise attenuation resulted in improved sleep hygiene and rate of growth.” Invictus Medical is a privately held company.

FDA 52

FDA Medical Recruitment Specialization 52

Referral MD

NOVEMBER 16, 2023

With eConsults, ReferralMD improves the patient experience, reduces costs, and expands the scope of primary care by providing crucial clinical guidance from specialists. Primary care providers can confer with specialists through eConsults before making referrals for face-to-face care. To learn more about ReferralMD, please visit [link].

Consulting 52

Consulting Networking Management CRM 52

Pharmaceutical Technology

NOVEMBER 9, 2022

On November 2, the Institute for Clinical and Economic Review (ICER) released its updated evidence aimed at measuring the clinical effectiveness and cost of the two haemophilia gene therapies. Known by the brand name Roctavian, BioMarin’s haemophilia A therapy valoctocogene roxaparvovec could be fairly priced in the range of $1.95–1.96

Safety 111

Safety Pharmaceutical Patients Medicine 111

Pharmaceutical Technology

JULY 14, 2022

AVR-RD-05 had previously obtained rare pediatric disease designation from the FDA. Next year, Avrobio is expected to initiate a collaborator-sponsored Phase I/II clinical trial for Hunter syndrome in partnership with the University of Manchester, UK.

Food and Drug Administration 52

Food and Drug Administration FDA Prescription Food 52

Clarify Health

MAY 18, 2023

Last September, CHI released “The Kids Are Not Alright: Pediatric Mental Health Care Utilization from 2016-2021,” which looked at mental health utilization among children with a diagnosed mental illness.

Media 52

Media Healthcare Healthcare Insurance 52

World of DTC Marketing

AUGUST 13, 2021

A TELLING SPIKE: Dr. Brynn Marks, a pediatric endocrinologist at Children’s National Hospital in Washington, D.C., Brynn Marks, a pediatric endocrinologist at Children’s National Hospital in Washington, D.C., …At Children’s National Hospital in Washington, D.C.,

Insurance 187

Insurance Medical Patients Management 187

Legacy MEDSearch

NOVEMBER 28, 2022

Based on customer feedback, the company designed the BodyTom 64 to enhance the user experience and improve clinical workflows through revisions to both the software and the data acquisition system (DAS). We offer a variety of different solutions for hiring managers depending on the scope and scale of each individual search.

FDA 92

FDA Food and Drug Administration Electronics Recruitment 92

Medgadget

OCTOBER 11, 2022

At Johns Hopkins, clinical researchers have developed a set of surgical tools that allow them to more easily create a replacement ear for those born with malformed or missing ears. This is difficult – surgeons are clearly very skilled with their hands, but artistic skills, such as sculpting, often are a little outside their comfort zone.

Medicine 115

Medicine Patients 115

Pharmaceutical Technology

FEBRUARY 19, 2023

STRO-002 is under clinical development by Sutro Biopharma and currently in Phase I for Refractory Acute Myeloid Leukemia. Attributes of the drug, company and its clinical trials play a fundamental role in drug-specific PTSR and likelihood of approval. It is administered through intravenous route.

Biopharma 40

Biopharma Manufacturing Pharmaceutical Medicine 40

Pharmaceutical Technology

MARCH 2, 2023

Rituximab biosimilar is under clinical development by Dr. Reddy’s Laboratories and currently in Phase III for Rheumatoid Arthritis. Attributes of the drug, company and its clinical trials play a fundamental role in drug-specific PTSR and likelihood of approval. The company also develops and markets generic biosimilar products.

Pharmaceutical 40

Pharmaceutical Marketing Manufacturing 40

Legacy MEDSearch

MARCH 22, 2023

Designates FibroScan as a non-invasive aid for the clinical management, diagnosis, and monitoring of adult and pediatric patients with confirmed or suspected liver disease as part of an overall assessment of the liver. ” Age has been removed as the first selection probe and exam-type step.

Management 52

Management FDA Food and Drug Administration Physicians 52