C-Path Opportunities Table
Rare and Orphan Diseases
Filter by Focus Area
Innovative Model-informed Drug Development Tools to Inform Trial Design in Neurological Diseases
Driving Regulatory Progress for Inherited Ataxias: CPTA’s Impact in Advocacy and Change
Inherited ataxias are a diverse collection of genetic disorders that are characterized by clinical manifestations like impaired coordination of voluntary movements, such as gait and speech.
United Against Cancer: John Galas’ Impact on Sarcoma and Soccer
John Galas has spent most of his life around soccer. At an early age, he developed a deep passion for the sport and embraced the competitive spirit inherent in it. This mindset that proved invaluable when he faced his toughest opponent yet: cancer.
C-Path Roundtable: Help4HD Unites to Discuss Advancements in Huntington’s Disease Treatments
From Diagnosis to Advocacy: Layne Oliff’s Unwavering Commitment to ALS Awareness and Action
Layne Oliff was an active runner who successfully completed 10-mile trail runs up until his 60th birthday — before he was diagnosed with amyotrophic lateral sclerosis (ALS) in November of 2020. Layne soon pivoted that same drive and commitment to another journey, as an active advocate in the ALS community.
Active Lifestyles Key for Parkinson’s Advocates Zenner-Dolan, Kwok as They Await Advancements in Treatments
Dr. Kevin Kwok and Sarah Zenner-Dolan are drawn together by more than their respective experiences living with Parkinson’s disease (PD). Both have stepped up as champions for those living with PD.
For Judy Ashley’s Family, ADPKD Impact Spans Generations, Sparks Advocacy Work in Community
Judy Ashley is one of four siblings living with PKD. The family lost their mother when she was just 61, due to complications from dialysis. All five family members and one of Ashley’s nieces received transplants. “My energy now goes to empowering my daughter to be her own advocate,” Ashley explained. “I keep telling her, if it doesn’t make sense, if you don’t agree with [the treatment plan] keep asking questions.”
Living with ARPKD Presents Challenges, Opportunities for Cormack Family
When Lisa Cormack’s daughter Jaina was 4 years old, she went to the pediatrician for a typical preschool physical. A healthy, happy kid, there was no reason to fear anything abnormal or concerning would be discovered as part of the checkup.
Given No Answers After a Life-Changing Diagnosis, Pat Furlong Worked to Create Her Own
C-Path’s Alzheimer’s Disease Simulator Featured in the Wall Street Journal
Addressing Critical Unmet Needs through the Critical Path for Alzheimer’s Disease
Alzheimer’s disease (AD) accounts for an estimated 60% to 80% of dementia cases 2 and total costs for AD care in the U.S. are estimated to increase to more than $1 trillion by 2050.
A call to action in Parkinson’s research and care: The benefits of sharing patient-level data
Alzheimer’s Patient Advocate Montminy Uses Voice to Influence Alzheimer’s Treatments, Sees Hope in Future Treatments
When Joe Montminy was just 54 years old, he began noticing changes that impacted his ability to do his job. Routine work soon had him working nights and weekends to compensate for decreased productivity.
By Bringing Patients’ Voices to the Forefront, Sarcoma Advocates Illustrate Community’s Innovative Impact
With such expansion, it is important to highlight some incredible advocates who are helping lead the effort on drug repurposing in advancing treatment options for sarcoma research.
CPTA — Impact Through Optimization of Clinical Trial Design
Drug Repurposing Provides Big Impact for Patients
C-Path's CDRC is positioned to inform future clinical trials for diseases of high unmet medical need with real-world clinical outcome data.
Duchenne Advocates Russell, Werth Use Platform to Amplify Patient Impact
How RDCA-DAP Delivers Impact and Support for Rare Disease Treatment Innovation
Impact For Patients: International Neonatal Consortium
INC’s work has created a paradigm shift in clinical drug development for neonatal seizures.
Impact For Patients: Critical Path for Parkinson’s Consortium
Impact For Patients: Patient-Reported Outcome Consortium
Patient-Reported Outcome (PRO) Consortium helps to provide drug developers with the clinical outcome assessment (COA) tools necessary to target symptoms and functional impacts that matter most to patients.
Impact For Patients: TTC’s Work to Qualify a Reasonably Likely Surrogate Endpoint
TTC’s work to qualify a reasonably likely surrogate endpoint will speed up drug development for kidney transplant patients.
Impact For Patients: TTC’s iBox Scoring System
TTC is working to qualify the iBox Scoring System with FDA as a reasonably likely surrogate endpoint (RLSE) for kidney transplant clinical trials.
Impact on Industry and Patients: Polycystic Kidney Disease Outcomes Consortium
C-Path as Convener, Curator, Innovator and Accelerator
Impact on Industry: Critical Path to TB Drug Regimens
The collaborative work of the CPTR Initiative has contributed to the development of the first new drugs for TB in over 40 years, and has solidified a drug regimen development pipeline in TB.
Technological Break Throughs Lead to Improved T1D Outcomes, But Still Work to Be Done for T1DC
As a person living with Type 1 Diabetes (T1D) who works to advance treatments for autoimmune diseases as a Drug Development Scientist for IGM Biosciences, Ryan Eberwine has seen a lot of progress since his diagnosis 24 years ago.
The Impact of C-Path’s Quantitative Medicine Program: Generating Cutting-edge Solutions to Accelerate Drug Development
As a global leader in regulatory science, the generation of actionable quantitative solutions to accelerate drug development lies at the heart of the Critical Path Institute's (C-Path) core competencies, led by the Institute's Quantitative Medicine (QuantMed) Program.