A new type of Charcot-Marie-Tooth (CMT) has recently been identified caused by mutations in the SORD gene.
Blog Category
Types of CMT
Total posts in Category: 1
Young Entrepreneur with CMT1A Launches Company
Dakota launched her own business, Blue Bear in August 2020 with a full line of greeting cards, stickers and enamel pins.
Jaxson’s Spooky Shoot 2020
Thank you to everyone who attended this family-friendly event with costumes. Check out the photo gallery!
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
The FDA has agreed with the key elements of Pharnext’s approach for the developmental pathway to approval for PXT3003.
Clinical Trial for CMT1A begins in Korea
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.
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
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
Pharnext raises € 7.7 million in a private placement
Pharnext SA (FR0011191287 – ALPHA) (the “Company”), a biopharmaceutical company pioneering a new approach to developing innovative drug combinations based on its PLEOTHERAPY artificial intelligence platform harnessing big genomics data and network pharmacology, today announced a capital raise of circa € 7.7 million by way of issuance of 1,799,061 new ordinary shares (the “New Shares”) with one warrant attached each (together with the New Shares, the “ABSA”).
Calling all Grandparents (and Parents)!
Join Iris Adler as she continues fundraising for her grandson and the many others living with Charcot-Marie-Tooth Type 2A
Why I’ll be attending the “Voice of the Patient Weekend” A Patient’s Perspective
I am attending HNF’s “Voice of the Patient” Weekend 2018 to tell the FDA and other stakeholders how TRPV4 HN impacts my life.
Pharnext’s lead PLEODRUG © for treatment of CMT1
Pharnext completed patient enrollment for the international Phase 3 clinical trial of PXT3003, Pharnext’s lead PLEODRUG ©, for treatment of CMT1A.
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Advocacy (7)
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CMT (2)
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CMT Type – CMT1A (22)
CMT Type – CMT1B (2)
CMT Type – CMT1X (3)
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CMT Type – CMT4A GDAP1 (3)
CMT Type – CNTNAP1 (1)
CMT Type – GAN (5)
CMT Type – HDAC6 (1)
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CMT Type – MTRFR-C12orf65 (6)
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Awareness (34)
Centers of Excellence (13)
CMT (2)
CMT en Español (1)
CMT Summit (53)
CMT Update (74)
CMT-Connect (29)
CMT-Connect Webinars (10)
Webinars – Accessible Travel (3)
Webinars – Adaptive Driving (2)
Webinars – Bracing (1)
Webinars – Breathing (1)
Webinars – Canine Companions (1)
Webinars – Cannabis (3)
Webinars – Dating and Relationships (3)
Webinars – Family Planning (1)
Webinars – FEATURED (6)
Webinars – Genetic Testing (1)
Webinars – Home Accessibility (2)
Webinars – Inclusive Employment (3)
Webinars – Inspire Community (1)
Webinars – Legal Rights and Benefits (3)
Webinars – Newly Diagnosed (2)
Webinars – Nutrition (1)
Webinars – Pain (5)
Webinars – Podcasts (1)
Webinars – PT and Exercise (9)
Webinars – Research and Clinical Trials (9)
Webinars – School and College (4)
Webinars – Surgery (2)
Webinars – Telemedicine (1)
Webinars- Emotional and Mental Health (5)
Community (4)
Emerging Technologies (1)
Faces of CMT (1)
Faces of CMT – Addie (2)
Faces of CMT – Bernadette (9)
Faces of CMT – CMT4A (7)
Faces of CMT – Grace (18)
Faces of CMT – HELP Fund (8)
Faces of CMT – HNPP Fund (3)
Faces of CMT – James (1)
Faces of CMT – Jaxson (7)
Faces of CMT – Zach (18)
Featured (28)
GRIN Patient Registry (39)
HNF Team (10)
Industry (3)
Living with CMT (78)
Medical professionals (2)
Movement Is Medicine (17)
Optic Hereditary Neuropathy (5)
Our Impact (3)
Clinical Trials (21)
PFDD (1)
PFDD Meeting 28SEP2018 (5)
Past Events (35)
Patience Resources (0)
Accessible Travel (4)
Bracing (11)
Breathing (3)
Canine Companions (5)
Cannabis (8)
Caregivers (3)
Dating and Relationships (7)
Emotional and Mental Health (9)
Family Planning (1)
Fashion and Products (1)
Genetic Testing (11)
Home Accessibility (2)
Inclusive Employment (3)
Inspire Community (7)
Legal Rights and Benefits (8)
Neurotoxic Drugs (4)
Newly Diagnosed (7)
Nutrition (3)
Occupational Thearpy (2)
Pain (8)
Podiatry (4)
PT and Exercise (25)
Research and Critical Trials (29)
School & College (6)
Surgery (10)
Telemedicine (1)
Tips and Hacks (2)
Patient-Focused Research (1)
Pediatrics & CMT (10)
Registry (16)
Research (95)
School Outreach Program and Team CMT Kids (1)
Special Events (2)
Bike New York (19)
Other Events (4)
Spin-a-thon (7)
TCS New York City Marathon (7)
Team CMT Members (61)
The CMT Genie (6)
TRIAD (28)
TRIAD – Clinical Trial Readiness (5)
Vitaccess (1)
TRIAD – Research Gifts (0)
University of Helsinki (2)
Veneto (1)
TRIAD – Sponsored Research (0)
Burke Insitute (3)
University of Cambridge (1)
University of Miami (1)
University of San Antonio Texas (1)
TRIAD – Therapeutics (4)
Applied Therapeutics (10)
Pharnext (10)
Rarebase (4)
Types of CMT (1)
CMT Type – CMT1A (22)
CMT Type – CMT1B (2)
CMT Type – CMT1X (3)
CMT Type – CMT2A (9)
CMT Type – CMT2C (5)
CMT Type – CMT4A GDAP1 (3)
CMT Type – CNTNAP1 (1)
CMT Type – GAN (5)
CMT Type – HDAC6 (1)
CMT Type – HNPP (3)
CMT Type – MTRFR-C12orf65 (6)
CMT Type – SORD Deficiency (9)
Upcoming Events (3)
Ways to Give (4)
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