Dr. Nicole Muschol, MD
Claim this profileUniversity of Hamburg
Studies Niemann-Pick Disease
Studies Sanfilippo Syndrome
4 reported clinical trials
5 drugs studied
Area of expertise
1Niemann-Pick Disease
NPC2 positive
NPC1 positive
2Sanfilippo Syndrome
Clinical Trials Nicole Muschol, MD is currently running
N-Acetyl-L-Leucine
for Niemann-Pick Disease Type C
A pivotal, randomized, double-blind, placebo controlled, multi-center therapeutic study for patients age 4 and older with a confirmed diagnosis of Niemann Pick disease type C (NPC). The objective of this study is to evaluate the safety, tolerability and efficacy of N-acetyl-L-leucine (IB1001) compared to standard of care. Following this randomized, double-blind, placebo-controlled "Parent Study", an extension phase is conducted for (1) patients who completed the "Parent Study" and (2) patients who are enrolled directly into the Extension Phase. Currently, the Extension Phase provides patients with 3 years of open-label treatment.
Recruiting1 award Phase 310 criteria
Tividenofusp Alfa
for Hunter Syndrome
This trial is testing a new treatment called tividenofusp alfa for patients with a rare genetic disorder called MPS II. The treatment aims to replace missing enzymes to help break down harmful substances in the brain and body.
Recruiting1 award Phase 2 & 33 criteria
More about Nicole Muschol, MD
Clinical Trial Related3 years of experience running clinical trials · Led 4 trials as a Principal Investigator · 2 Active Clinical TrialsTreatments Nicole Muschol, MD has experience with
- N-Acetyl-L-Leucine
- AX 250
- DNL310
- Idursulfase
- FLT201
Breakdown of trials Nicole Muschol, MD has run
Niemann-Pick Disease
Sanfilippo Syndrome
Sanfilippo syndrome
Hunter Syndrome
Sudden Infant Death Syndrome
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Frequently asked questions
Do I need insurance to participate in a trial?
Almost all clinical trials will cover the cost of the ‘trial drug’ — so no insurance is required for this. For trials where this trial drug is given alongside an already-approved medication, there may be a cost (which your insurance would normally cover).
What does Nicole Muschol, MD specialize in?
Nicole Muschol, MD focuses on Niemann-Pick Disease and Sanfilippo Syndrome. In particular, much of their work with Niemann-Pick Disease has involved NPC2 positive patients, or patients who are NPC1 positive.
Is Nicole Muschol, MD currently recruiting for clinical trials?
Yes, Nicole Muschol, MD is currently recruiting for 2 clinical trials in the USA. If you're interested in participating, you should apply.
Are there any treatments that Nicole Muschol, MD has studied deeply?
Yes, Nicole Muschol, MD has studied treatments such as N-Acetyl-L-Leucine, AX 250, DNL310.
What is the best way to schedule an appointment with Nicole Muschol, MD?
Apply for one of the trials that Nicole Muschol, MD is conducting.
Is there any support for travel costs?
The coverage of travel expenses can vary greatly between different clinical trials. Please see more financial detail in the trials you’re interested to apply.
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