Helixmith, specializing in gene therapy research for over 20 years, has kicked off its phase I and 2a clinical trial for using VM202 (Engensis) to treat CMT1A.
Blog Archive
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The Miracle We’ve Been Praying For!
Alana Kohler’s story of having CMT4A.
University of North Carolina Becomes New Center of Excellence
Introducing Dr. Rebecca Traub, University of North Carolina
HEREDITARY NEUROPATHY FOUNDATION (HNF) IS HERE FOR YOU DURING THIS TIME OF UNCERTAINTY
HNF team has been working tirelessly to produce content and programs to help the CMT Community during this time of uncertainty.
Microcirculation
Improve blood flow and learn about this holistic approach to increase oxygen and decrease toxins.
Press Release: Pharnext provides regulatory and clinical update on PXT3003 Phase III study for the treatment of Charcot-Marie-Tooth Type 1A
US Food and Drug Administration has agreed with Pharnext and provided clear guidance on the regulatory pathway to approval for PXT3003, including key design elements of a single pivotal Phase III study
HNF’s Newest Center of Excellence: Austin Neuromuscular Center, Dr. Yessar Hussain
Introducing Yessar Hussain
Good News for CMT1A Patients – PXT3003
Three major regulatory agencies in the United Kingdom, Europe and US have recognized PXT3003 as a lead drug candidate to treat CMT1A.
Novel findings of a new common type of CMT2 that might be the 1st step to a treatment: SORD gene deficiency, the most common autosomal-recessive type of CMT
a mutation in the SORD gene that may may affect 60,000 patients worldwide
Painting By Mouth – Kaileen Selig
CMT doesn’t stop artist, Kaileen Selig from painting beautiful pieces using her mouth.
GRIN powers HNF’s patient-centered research and drug discovery initiatives.
GRIN has played a critical role in identifying important issues related to the CMT patient experience, revealing new areas to explore and research.
Meet Dr. Nivedita Jerath, MD, MS – AdventHealth Neuroscience Institute Center of Excellence
The AdventHealth Neuroscience Institute is fully committed to patient excellence, care, and exceeding expectations. Patients will experience a team that is superb beyond expectations. If they are coming from far, they will be catered to with our destination team. When they arrive, they will experience an environment full of love, kindness, friendliness, and softness. They will observe an amazing facility, receive an in-depth physician evaluation, electrodiagnostic testing, genetic testing, and opportunities to get all the multidisciplinary therapies that they need. They will also be involved in any research studies that we may have.
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