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April 3, 2020April 3, 2020

With $30M Investment, Regenacy Plans Phase 2 Trial Testing Ricolinostat for CMT Type 2

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Regenacy Pharmaceuticals received $30 million to help advance its lead candidate ricolinostat into Phase 2 studies for Charcot-Marie-Tooth (CMT) disease type 2, as well as diabetic and chemotherapy-induced peripheral ... Read more

March 31, 2020March 31, 2020

NORD Partners with ‘Sing Me a Story’ for July 18–20 Living Rare Forum

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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 of the coronavirus disease COVID-19 pandemic. ... Read more

March 27, 2020March 27, 2020

PXT3003 Designated Promising Innovative Medicine in UK for CMT1A Treatment

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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) aged 16 and older. PIM ... Read more

March 20, 2020March 20, 2020

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

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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 27, 2020

Pharnext Raises 7.7M Euros to Develop PXT3003 Therapy for CMT1A

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France-based Pharnext will use a capital raise of about 7.7 million euros ($8.4 million) to support the clinical development of PXT3003, the company’s experimental treatment for Charcot-Marie-Tooth disease type ... Read more

March 17, 2020March 17, 2020

Mutation in SEPT9 Gene Can Lead to CMT, Report Says

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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 13, 2020March 13, 2020

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

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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

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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

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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

March 6, 2020March 6, 2020

HNF Brings ‘Movement is Medicine’ Summit to Florida

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To promote the value of exercise in Charcot-Marie-Tooth disease, the Hereditary Neuropathy Foundation is bringing its latest summit to central Florida. The free Movement is Medicine Summit Orlando will ... Read more

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