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CMT Awareness Month 2022
…with the disease, raising awareness is imperative. The CMTA is a patient-led nonprofit that is aggressively searching out a treatment for CMT by putting together a group of top-flight researchers…
Diana Bharucha-Goebel, MD Joins the CMTA-STAR Advisory Board
…is also interested in better understanding how we efficiently and safely target the peripheral nervous system with viral mediated gene transfer treatment approaches, studying total vector genome dose, parallel preventative…
Finding Your CMT Gene
Finding Your CMT Gene – The Distant Cousin Project The effort to identify genes that cause rare diseases is more important than ever since effective treatments for genetically caused rare…
2021 STAR Accomplishments
…the body responds to a treatment for a disease) Initiatives in CMT1A, CMT1X, CMT2A, CMT1B and others in process: INC progress in developing muscle MRI and blood sample measurements of…
CMT Type 2
…the function of the working protein and somehow treatment will need to silence that abnormal copy or cut it out. If it can be silenced, blocked or cut it out…
Keep Hope Alive for Everyone Living with CMT
…treatments are found to prevent/stop progression of CMT and that a cure will soon be within reach. But hope is more than just optimism: it is based on an expectation…
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…
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….
How to Contact Your Hometown Media
…be identified for early treatment. Charcot-Marie-Tooth is a progressive disorder of the peripheral nerves that causes people to lose the normal use of their feet and legs. Symptoms include: foot…