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Tagged: rare diseases

PSP Call for Papers: Model-Based Innovations in Pediatric Rare Disease

November 18, 2024

Join PSP Associate Editors Ping Zhao and Rajat Desikan as well as former Editor-in-Training Jane Knöchel as they discuss the journal’s latest Call for Papers “Model-Based Innovations in Pediatric Rare Disease.” Find out why the PSP Editorial Team chose to highlight this topic in 2025, what types of submissions they’re hoping to receive, why publishing in a themed issue can boost the profile of your research, and more!

ASCPT Podcast Channel
ASCPT Podcast Channel
PSP Call for Papers: Model-Based Innovations in Pediatric Rare Disease
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Download file | Play in new window | Duration: 8:53 | Recorded on November 18, 2024

Tagged With: Call for Papers, Pediatric Pharmacology, PSP, rare diseases, Special IssueChannel: ASCPT Podcast Channel, ASCPTPod

CPT Rare Diseases Call for Papers

May 13, 2024

Clinical Pharmacology & Therapeutics has an open call for papers on Rare and Orphan Diseases, but the deadline is closing soon! Check out the latest episode of ClinPharmPod, where Editors-in-Training Drs. Mai Mehanna and Ya-Feng (Jay) Wen detail why CPT chose this theme and what types of papers the editors are hoping to receive. If you have questions about submitting papers for this themed issue, please contact Managing Editor Aletha Gerding at [email protected].

ClinPharmPod
ClinPharmPod
CPT Rare Diseases Call for Papers
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Download file | Play in new window | Duration: 12:03 | Recorded on May 13, 2024

Tagged With: Call for Papers, CPT, Orphan diseases, rare diseasesChannel: ClinPharmPod

Sharing Data to Improve Patient-Focused Outcomes

April 26, 2022

Critical Path Institute’s Dr. Diane Stephenson joins host Alaina Webster to discuss the importance of data sharing to improve patient care, particularly in Neuroscience. Diane and her co-authors recently published a Review, “Can Innovative Trial Designs in Orphan Diseases Drive Advancement of Treatments for Common Neurological Diseases?”, in the April Neuroscience-themed issue of Clinical Pharmacology & Therapeutics, and she ties this work into successful data sharing models and their impact on patient outcomes. Critical Path Institute has accumulated more than 300 clinical data studies across 15+ disease areas, and its repository of data currently includes information on the following neuroscience diseases: Alzheimer’s disease, Huntington’s disease, ataxias, Duchenne muscular dystrophy, Parkinson’s disease, and multiple sclerosis.

ClinPharmPod
ClinPharmPod
Sharing Data to Improve Patient-Focused Outcomes
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Download file | Play in new window | Duration: 14:50 | Recorded on April 25, 2022

Tagged With: ASCPT, Best Practices, citizen science, clinical trial design, CPT, crowd sourcing, data sharing, Database, Neuroscience, Parkinson's disease, patient advocacy, rare diseasesChannel: ClinPharmPod

What’s New in Neuroscience?

March 19, 2022

Clinical Pharmacology & Therapeutics recently published its second themed issue of 2022 – Therapeutics in Neuroscience. Join Editor-in-Chief Dr. Piet van der Graaf and Associate/Guest Editors Drs. Spiros Vamvakas, Paulien Ravenstijn, and Iris Minichmayr as they delve into reasons for the theme, what papers caught their eyes, and what action items they hope to leave with readers. If you’re interested in submitting your research for any upcoming themed issues in ASCPT’s Journal Family, you can find Calls for Papers and more information here.

ClinPharmPod
ClinPharmPod
What’s New in Neuroscience?
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Download file | Play in new window | Duration: 13:07 | Recorded on March 18, 2022

Tagged With: Alzheimer's Disease, Amyotrophic Lateral Sclerosis, ASCPT, Biomarkers, Clinical Pharmacology and Therapeutics, ClinPharmPod, CPT, Food and Drug Administration, Gene Therapy, Huntington's Disease, Neuroscience, rare diseasesChannel: ClinPharmPod

Marlene Haffner on Orphan Drugs

November 28, 2016

This episode features Marlene Haffner, MD, MPH of Haffner Associates, LLC discussing orphan drug regulation in the United States and beyond. Read her article which published in the October 2016 issue here.

Listen and subscribe for free on iTunes and Google Play

ClinPharmPod
ClinPharmPod
Marlene Haffner on Orphan Drugs
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Download file | Play in new window | Duration: 19:50

Tagged With: ASCPT, Clinical Pharmacology and Therapeutics, Orphan drugs, rare diseases, small patient populationsChannel: ClinPharmPod

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