March 24, 2020March 23, 2020
Like Many, My Feelings About the COVID-19 Craziness Are Complicated
Columns, On My Own Two by Young Lee
Hello, I hope you are well. It’s a difficult time for many of us no matter where we live, ... Read more
March 23, 2020March 20, 2020
Managing My Anxiety During the COVID-19 Pandemic
CMT and Me - a Column by Jill Price, Columns
Are you feeling anxious about COVID-19 and its effects around the world? You are not alone. In these times ... Read more
March 20, 2020March 20, 2020
Global Rare Disease Group’s Focus: 1,000 New Therapies by 2027, Despite COVID-19
News
Even with the coronavirus pandemic ravaging Europe and much of the world, patient advocate Lucia Monaco, PhD, of Italy ... Read more
March 20, 2020March 20, 2020
Pharnext Raises 7.7M Euros to Develop PXT3003 Therapy for CMT1A
News
France-based Pharnext will use a capital raise of about 7.7 million euros to support the clinical development of PXT3003, ... Read more
March 17, 2020March 17, 2020
Mutation in SEPT9 Gene Can Lead to CMT, Report Says
News
A mutation in the SEPT9 gene — which provides instructions for making the septin-9 protein that is involved in ... Read more
March 17, 2020March 17, 2020
It’s Time for Disabled Narratives to Take the Spotlight
Columns, On My Own Two by Young Lee
As an Asian American, I shared the joy and pride felt by much of the global Asian community when ... Read more
March 16, 2020March 12, 2020
Having a Child Was the Right Decision for Me
CMT and Me - a Column by Jill Price, Columns
“Would you have had children if you knew they would have Charcot-Marie-Tooth?” I have heard this question before from ... Read more
March 13, 2020March 13, 2020
Acceleron Halts Development of ACE-083 for CMT Based on Phase 2 Trial Data
News
Acceleron Pharma is discontinuing the clinical development of ACE-083, its experimental treatment candidate for Charcot-Marie-Tooth (CMT) disease, based on ... Read more
March 10, 2020March 10, 2020
JAG1 Gene Mutations May Lead to Peripheral Nerve Damage Linked to CMT2, Study Reports
News
Mutations in the Jagged1 (JAG1) gene may compromise the integrity of peripheral nerves — those found outside the brain ... Read more
March 10, 2020March 10, 2020
Pediatric Rare Disease Therapies Increase, But Most Repurposed, Study Finds
News
The number of treatments for children with rare diseases has grown over the past decade, according to a new study. ... Read more
March 6, 2020March 6, 2020
HNF Brings ‘Movement is Medicine’ Summit to Florida
News
To promote the value of exercise in Charcot-Marie-Tooth disease, the Hereditary Neuropathy Foundation is bringing its latest summit to ... Read more
March 5, 2020March 5, 2020
Video: BioNews’ Social Media Campaign Highlights #WhatMakesMeRare
News
In recognition of Rare Disease Day Feb. 29, BioNews Services launched a social media campaign last month asking patients ... Read more

March 24, 2020March 23, 2020

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 spread to most of the ... Read more

March 20, 2020March 20, 2020

Global Rare Disease Group’s Focus: 1,000 New Therapies by 2027, Despite COVID-19

News

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 nonprofit she chairs will see ... Read more

March 20, 2020March 20, 2020

Pharnext Raises 7.7M Euros to Develop PXT3003 Therapy for CMT1A

News

France-based Pharnext will use a capital raise of about 7.7 million euros to support the clinical development of PXT3003, the company’s experimental treatment for Charcot-Marie-Tooth disease type 1A (CMT1A). ... Read more

March 17, 2020March 17, 2020

Mutation in SEPT9 Gene Can Lead to CMT, Report Says

News

A mutation in the SEPT9 gene — which provides instructions for making the septin-9 protein that is involved in many cell functions, including cell division and movement — can ... Read more

March 17, 2020March 17, 2020

It’s Time for Disabled Narratives to Take the Spotlight

Columns, On My Own Two by Young Lee

As an Asian American, I shared the joy and pride felt by much of the global Asian community when “Parasite,” a South Korean genre-bending thriller directed by Bong Joon ... Read more

March 16, 2020March 12, 2020

Having a Child Was the Right Decision for Me

CMT and Me - a Column by Jill Price, Columns

“Would you have had children if you knew they would have Charcot-Marie-Tooth?” I have heard this question before from friends and acquaintances when I told them about Charcot-Marie-Tooth disease ... Read more

March 13, 2020March 13, 2020

Acceleron Halts Development of ACE-083 for CMT Based on Phase 2 Trial Data

News

Acceleron Pharma is discontinuing the clinical development of ACE-083, its experimental treatment candidate for Charcot-Marie-Tooth (CMT) disease, based on recent data from a Phase 2 trial. While the trial ... Read more

March 10, 2020March 10, 2020

JAG1 Gene Mutations May Lead to Peripheral Nerve Damage Linked to CMT2, Study Reports

News

Mutations in the Jagged1 (JAG1) gene may compromise the integrity of peripheral nerves — those found outside the brain and spinal cord — and lead to peripheral neuropathy associated ... Read more

March 10, 2020March 10, 2020

Pediatric Rare Disease Therapies Increase, But Most Repurposed, Study Finds

News

The number of treatments for children with rare diseases has grown over the past decade, according to a new study. However, despite the increase, nearly 7,000 rare diseases are still ... Read more