NORD Partners with ‘Sing Me a Story’ for July 18–20 Living Rare Forum
The Living Rare, Living Stronger Patient and Family Forum, originally set for May 14–16 in Cleveland, Ohio, has been postponed until July 18–20 because ... Read more
Updates
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PXT3003 Designated Promising Innovative Medicine in UK for CMT1A Treatment
The U.K.’s Medicines and Healthcare products Regulatory Agency (MHRA) has granted promising innovative medicine (PIM) designation to PXT3003 for the treatment of people with Charcot-Marie-Tooth type 1A (CMT1A) ... Read more -
Like Many, My Feelings About the COVID-19 Craziness Are Complicated
Hello, I hope you are well. It’s a difficult time for many of us no matter where we live, as the COVID-19 virus has ... Read more -
Managing My Anxiety During the COVID-19 Pandemic
Are you feeling anxious about COVID-19 and its effects around the world? You are not alone. In these times of uncertainty, it’s natural to ... Read more -
Global Rare Disease Group’s Focus: 1,000 New Therapies by 2027, Despite COVID-19
Even with the coronavirus pandemic ravaging Europe and much of the world, patient advocate Lucia Monaco, PhD, of Italy remains confident that the Paris-based ... Read more
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Pharnext Raises 7.7M Euros to Develop PXT3003 Therapy for CMT1A
France-based Pharnext will use a capital raise of about 7.7 million euros ($8.4 million) ... -
Mutation in SEPT9 Gene Can Lead to CMT, Report Says
A mutation in the SEPT9 gene — which provides instructions for making the septin-9 ... -
It’s Time for Disabled Narratives to Take the Spotlight
As an Asian American, I shared the joy and pride felt by much of ... -
Having a Child Was the Right Decision for Me
“Would you have had children if you knew they would have Charcot-Marie-Tooth?” I have ... -
Acceleron Halts Development of ACE-083 for CMT Based on Phase 2 Trial Data
Acceleron Pharma is discontinuing the clinical development of ACE-083, its experimental treatment candidate for ... -
JAG1 Gene Mutations May Lead to Peripheral Nerve Damage Linked to CMT2, Study Reports
Mutations in the Jagged1 (JAG1) gene may compromise the integrity of peripheral nerves — ...
Despite Functional Limitations, Few CMT Patients Receive Physical and Occupational Therapy
News
Most people with Charcot-Marie-Tooth (CMT) disease do not receive physical or occupational therapy, despite having extensive problems with physical functioning that limit mobility and quality of life, according to a report presented at the American Association of Neuromuscular & ... Read more