Drug Trials Snapshots: AUSTEDO

… a drug used for the treatment of chorea in patients with Huntington’s disease. Chorea is a condition characterized by uncontrolled, … and is one of many symptoms that occur in patients with Huntington’s disease. How is this drug used? AUSTEDO is a …

Orphan Drug Act - Relevant Excerpts

… (1) there are many diseases and conditions, such as Huntington's disease, myoclonus, ALS (Lou Gehrig's disease), Tourette … … There are many diseases and conditions, such as Huntington's disease, myoclonus, ALS (Lou Gehrig's disease), …

Huntington's Disease PFDD Meeting Slides

… Public Meeting on Huntington’s Disease Patient-Focused Drug Development September 22, 2015 1 … Drug Development Initiative – Background on Huntington’s Disease and Therapeutic Options – Overview of …

Transcript for PFDD for Huntington's Disease

… 3 1 C O N T E N T S 2 AGENDA ITEM: PAGE 3 MORNING SESSION: Huntington's Disease 4 SOUJANYA GIAMBONE, Introduction 5 5 WILLIAM DUNN, … T E N T S (Cont'd) 2 AGENDA ITEM: PAGE 3 MORNING SESSION: Huntington's Disease 4 PUJITA VAIDYA, Facilitator, public …

Huntington's Disease: Voice of the Patient

… (FDA’s) Patient-Focused Drug Development Initiative Huntington’s Disease Public Meeting: September 22, 2015 Report Date: March … 6 Overall impact of Huntington’s Disease on daily life …

Agenda for PFDD on Huntington's Disease

… Huntington’s Disease Public Meeting on Patient-Focused Drug Development … PhD Director, OSP, CDER, FDA 9:20 – 9:30 am Background on Disease Area and Treatment Dave Podskalny, MD Medical … 2: Patient perspectives on current approaches to treating Huntington’s Disease. A panel of patients and patient …

The Voice of the Patient: Huntington’s Disease

… (FDA’s) Patient-Focused Drug Development Initiative Huntington’s Disease Public Meeting: September 22, 2015 Report Date: March … 6 Overall impact of Huntington’s Disease on daily life …

The Path Forward: Advancing Treatments and Cures for Neurodegenerative Diseases - 07/29/2021

… to testify before you today on neurodegenerative disease drug development and approval. I have spent a … FDA approved novel therapies for patients with Parkinson’s disease, multiple sclerosis, spinal muscular atrophy, … neurodegenerative diseases including Friedreich’s ataxia, Huntington’s disease, Lewy body dementia, Parkinson’s …

The Story Behind the Orphan Drug Act

… The relatively limited prevalence of a particular disease acted as a barrier for commercial investment in the … and advocates affected by such rare diseases as Gaucher’s disease, Tourette’s syndrome, Huntington’s disease, severe combined immunodeficiency …

Upcoming EL-PFDD Meetings

… transparency and communication, FDA is sharing a list of disease areas where a letter of intent (LOI) has been … 2024 Pre-symptomatic and Early to Mid-stage Adult Onset of Huntington’s Disease (HD) Huntington’s Disease Society of America (HDSA) Phyllis …

Digital Health Technologies for Drug Development: Demonstration Projects

… projects on DHTs: Measuring Digital Clinical Endpoints in Huntington's Disease (MEND-HD) Awardee : University of Rochester Project … and continuously using wearable sensors, in early-stage Huntington’s disease (HD), a rare, progressive …

Patient Listening Session Summaries

… March 20, 2023 Carcinoid Syndrome – April 27, 2023 Celiac Disease - February 20, 2019 Childhood Cerebral Adrenal … (HCU) External Link Disclaimer  - June 26, 2020 Huntington's Disease Pre-Symptomatic Population External Link … (JDM) (summary unavailable) - March 16, 2021 Juvenile Huntington’s Disease (JHD) External Link Disclaimer – …

Division of Pharmacometrics

… Guidances and Presentations Publications Model/Data Format Disease Specific Model Library   [Archived] Program of … 4 Diabetes disease model [5] Clinical trial design 5 Huntington’s disease model [6] Patient enrichment and …   W Sun, D Zhou, J Warner, D Langbehn, G Hochhaus, Y Wang. Huntington's Disease progression: A population modeling …

Office of Neuroscience - Division of Neurology I

… and neuromuscular disorders, such as Alzheimer’s disease, Parkinson’s disease, Huntington’s disease, muscular dystrophy, amyotrophic lateral …

Condition-Specific Meeting Reports and Other Information Related to Patients' Experience

… an endorsement of their authors, sponsors, or content.   Disease or Condition (alphabetical) Type of Meeting … Listening Session Summary January 30, 2023 Juvenile Huntington’s Disease (JHD) Patient Listening Session Patient … Session Patient Listening Session Summary July 19, 2022 Huntington’s Disease (HD) Pre-Symptomatic Population Patient …

DDT COA #000129: Advanced Gait Analysis

… DDT COA #000129 Instrument Name Advanced Gait Analysis Disease/Condition Huntington’s Disease Concept of Interest Progressive gait abnormality …

Office of the Chief Medical Officer

… newborns) and patients with rare diseases (e.g., ALS and Huntington’s disease).  Enhance the clarity, predictability, efficiency, …

Marc Allard, PhD, Research Microbiologist

… one of the scientists who discovered the gene that causes Huntington’s disease. What made you choose to work for the U.S. Food and … genetic sequences of hundreds of thousands of foodborne disease-causing bacteria. Scientists from state, federal, and …

Olivia's Healing Herbs

… for the treatment, cure, mitigation, or prevention of disease.  For example, your website recommends or suggests … radiation poisoning, influenza, hepatitis, epilepsy, Huntington’s disease, and malaria. As explained further below, the …

Root Bioscience Brands, LLC dba Naternal - 673646 - 08/30/2024

… diagnosis, cure, mitigation, treatment, or prevention of disease, and/or intended to affect the structure or any … disease, in addition to protecting neurons in animals with Huntington's disease.” “On the cancer front, CBG has been … mice showed that CBG provided neuroprotection related to Huntington’s disease. Furthermore, CBG may inhibit the growth …

Scientific Public Private Partnerships and Consortia

… Health Technologies Project Critical Path for Alzheimer's Disease (CPAD) Critical Path for Parkinson’s (CPP) Critical Path for Sickle Cell Disease (CP-SCD) Critical Path to TB Drug Regimens Consortium … Board on Clinical Trials Heart Failure Collaboratory Huntington’s Disease Regulatory Science Consortium (HD-RSC) …

Letter of Support (LOS)

… assay As a monitoring biomarker, indicating progression of Huntington’s Disease in patients bearing the HD mutation 1/17/19: Letter … Biomarker Panel for use in Early Stage Diabetic Kidney Disease  6/14/2016: Letter of Support (PDF)  Harald Mischak …

Amerigo Labs LLC - 543052 - 04/04/2018

… use in the cure, mitigation, treatment, or prevention of disease. As explained further below, introducing or … ginkgo biloba as a medicinal herb for treating Alzheimer’s disease…” vi.     Under ingredient, glycine: “Major … of memory loss, schizophrenia, Parkinson’s disease, Huntington’s disease...”   Your products are not generally …

How FDA Plans to Help Consumers Capitalize on Advances in Science

… understanding of the genetic and protein bases of human disease. These developments are already being translated into … to develop such natural history models in Parkinson’s disease, Huntington’s disease, Alzheimer’s disease, and muscular …

Sovereign Laboratories, LLC - 566227 - 02/05/2019

… use in the cure, mitigation, treatment, or prevention of disease. As explained further below, introducing or … the inflammatory processes that precede Alzheimer’s disease. Studies have shown PRPs improve the cognitive … of Alzheimer’s disease, along with the symptoms of Huntington’s disease, sporadic ALS, epilepsy, post-stroke …