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CMT2D Research
…Scientific Advisory Board, is using mouse models of several forms of CMT to determine which types may be candidates for treatment with HDAC6 inhibitors and whether HDAC6 inhibitors may be…
New Mobile App Simplifies Sign-Up for Patient Registry
…treatment, can be found at www.cmtausa.org/research/cmt-clinical-trial-registry/. Signing up for the registry gives the user access to a list of current open studies and contact information for signing up for them….
NJ/NYC Focus Group with Acceleron Pharma
…furthering the development of treatments, and ultimately a cure, for CMT. One of the first CMTA Patients as Partners in Research projects was a focus group coordinated in partnership with…
CMTA and Addex Announce CMT1A Collaboration
CMTA and Addex Therapeutics Enter Collaboration to Advance GABA B PAM as Potential Treatment for CMT1A Addex Therapeutics is a clinical-stage pharmaceutical company pioneering allosteric modulation-based drug discovery and development….
CMTA and Taysha Gene Therapies Announce Collaboration for CMT4A
…Gene Therapies (Nasdaq: TSHA), a patient-centric, pivotal-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS)…
Pharnext Premier PXT3003 for CMT1A Phase 3 Study
…for CMT1A, and treatment consists of supportive care such as orthoses, physical and occupational therapy or surgery. The purpose of the PREMIER study is to discover if PXT3003 is effective…
Seattle Focus Group with Acceleron Pharma
…the work of our Strategy to Accelerate Research (STAR) and showcase ways the patient community can participate in furthering the development of treatments, and ultimately a cure, for CMT. As…
The CMTA Announces the Funding of a New Research Study for CMT2A
…(CMT2A). The aim of the study is to develop a night-time breathing treatment similar to CPAP (for sleep apnea) with the goal of helping respiratory issues and improving breathing for…
Terms & Conditions
…or treatment. The CMTA does not guarantee the accuracy, adequacy or completeness of any information and is not responsible for any errors or omissions or for the results obtained from…
The Summer 2023 CMTA Report
…of interest: CMT Patients Urge Faster Clinical Trials Applied Therapeutics Research Update DTx Treatment for CMT1A Under the Microscope: ASOs CMTA Funds Preclinical Work on CMT1B Mice Summer with CMT…