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Sperm Donor with Gene for Charcot-Marie-Tooth Disease Passes It on to Multiple Families
…and patients to accelerate scientific breakthroughs. The CMTA also offers community services, including 70 local branches, educational materials and patient conferences. To learn more about the CMTA, please visit www.cmtausa.org….
The CMTA Earns a Perfect Rating From Charity Navigator
…commitment to maintaining the highest levels of accountability and transparency.” For 19 years, Charity Navigator has rated over 195,000 charities. Charity Navigator evaluates nonprofit organizations’ financial health, including measures of…
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
…websites of local TV and radio stations, community newspapers, etc. Many will also provide guidelines for submitting stories and information about events, and the sample press release below will also…
Research Project: CMT Biomarkers and Outcome Measures
…over a one-year period. You may also combine this with your annual trip to the CMT clinic and continue your participation in the Inherited Neuropathy Consortium (INC) (https://www.rarediseasesnetwork.org/cms/inc) if already…
CMTA Camp Footprint Has Gone Virtual for 2020
…is building, and the countdown has begun. While registration for Camp Footprint 2020 is full, we’re so excited to welcome you to join us next year! Learn more at www.cmtacamp.org….
Acceleron Announces Topline Results from the Phase 2 Trial of ACE-083 in Patients with Charcot-Marie-Tooth Disease
ACE-083 did not achieve statistically significant improvements in functional endpoints relative to placebo; Acceleron to discontinue development of ACE-083. CAMBRIDGE, Mass.–(BUSINESS WIRE)– Acceleron Pharma Inc. (NASDAQ:XLRN), a biopharmaceutical company dedicated…
CMTA Funds Cutting-Edge Gene Editing Studies for Type 2 CMT
…cost, accuracy and efficiency of gene editing. Drs. Conklin and Judge, leaders in genome editing, and their team (www.ConklinLab.org) have already made substantial progress in testing CRISPR in stem cell…
Type 1 CMT Gene Therapy Project Now Underway
…their behalf. To learn more about the CMTA, please visit www.cmtausa.org. Media contacts: Mary Fiance Director, Public Relations and Communications, MDA (646) 992-2908 mfiance@mdausa.org Marcia Semmes Media Relations, CMTA marcia@cmtausa.org…