ウェーブ・ライフサイエンス社、ハンチントン病におけるアレル選択的変異型ハンチンチン低下作用を臨床的に初めて実証したフェーズ1b/2a SELECT-HD試験の良好な結果を発表

Today, Wave Life Sciences announced positive results from SELECT-HD, our Phase 1b/2a placebo-controlled trial evaluating the investigational therapy WVE-003. These results demonstrate that WVE-003 selectively lowers toxic, mutant huntingtin (mHTT) […]
PTC は本日、経口 PTC518 の第 2 相 PIVOT-HD 試験の有望な 12 ヵ月結果を発表した。

At Month 12, mutant Huntingtin (mHTT) in the blood was lowered by 22% and 43% respectively for 5mg and 10mg doses. A similar result was seen in cerebrospinal fluid, where […]
セージ・セラピューティクス社の第2相試験でハンチントン病の認知機能への影響が確認される

Earlier this week Sage Therapeutics announced the results of their SURVEYOR study which quantified the cognitive impact of Huntington’s Disease. Using the HD-Cognitive Assessment Battery (HD-CAB) they measured the difference […]
中東・北アフリカからの最新情報

IHA President Svein Olaf Olsen was keynote speaker on Huntington’s Disease at the MENA Congress for Rare Diseases in Abu Dhabi last month. He spoke of the urgent need to […]
FDAがuniQure社に再生医療アドバンスセラピー(RMAT)の指定を与える

Today we are very happy in the Huntington community. The announcement from Uniqure that the company has received the Regenerative Medicine Advanced Therapy (RMAT) “Designation for Investigational Gene Therapy AMT-130 […]
私たちは存在するタニタ・アレン、米国で遺伝子検査を受けるための闘いを語る

タニタ・アレンは回顧録『We Exist』の中で、自分の症状をハンチントン病と正しく診断してもらうのに苦労したことを語っている。何度も何度も誤診された。
HD患者のための無料オンライン心理療法プログラム

Doctors at the University of Reading (UK) have developed a free online psychotherapy programme for people with Huntington’s Disease. It’s part of a research project that looks at how people […]
パキスタンでハンチントン病協会が設立される - HDSOP

Founding of the Huntington’s Disease Society of Pakistan April 24th 2024 Last week another national association was created. The Huntington’s Disease Society of Pakistan (HDSOP) joined the growing list of […]
SOMバイオテック社、ハンチントン病における舞踏病治療薬に関する第IIb相試験の募集を完了

独自の人工知能プラットフォーム(SOMAIPRO®)を基盤とする臨床段階の創薬開発企業であるSOMバイオテック社は、臨床第IIb相 [...]...
プリレニア社、ハンチントン病治療薬プリドピジンのEUにおける販売承認申請を予定

Prilenia announced today that they plan to submit a Marketing Authorization Application in the EU for the use of Pridopidine in the treatment of Huntington’s Disease. According to Dr. Michael […]