JPWO2022251208A5 - - Google Patents

Download PDF

Info

Publication number
JPWO2022251208A5
JPWO2022251208A5 JP2023572721A JP2023572721A JPWO2022251208A5 JP WO2022251208 A5 JPWO2022251208 A5 JP WO2022251208A5 JP 2023572721 A JP2023572721 A JP 2023572721A JP 2023572721 A JP2023572721 A JP 2023572721A JP WO2022251208 A5 JPWO2022251208 A5 JP WO2022251208A5
Authority
JP
Japan
Prior art keywords
patient
viral vector
administered
optionally
weeks
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
JP2023572721A
Other languages
English (en)
Japanese (ja)
Other versions
JP2024520411A (ja
JP2024520411A5 (https=
Publication date
Application filed filed Critical
Priority claimed from PCT/US2022/030716 external-priority patent/WO2022251208A1/en
Publication of JP2024520411A publication Critical patent/JP2024520411A/ja
Publication of JP2024520411A5 publication Critical patent/JP2024520411A5/ja
Publication of JPWO2022251208A5 publication Critical patent/JPWO2022251208A5/ja
Pending legal-status Critical Current

Links

JP2023572721A 2021-05-24 2022-05-24 Ｘ連鎖性ミオチュブラーミオパチーの改善された治療のための組成物及び方法 Pending JP2024520411A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202163192279P 2021-05-24 2021-05-24
US63/192,279 2021-05-24
US202163245611P 2021-09-17 2021-09-17
US63/245,611 2021-09-17
PCT/US2022/030716 WO2022251208A1 (en) 2021-05-24 2022-05-24 Compositions and methods for improved treatment of x-linked myotubular myopathy

Publications (3)

Publication Number Publication Date
JP2024520411A JP2024520411A (ja) 2024-05-24
JP2024520411A5 JP2024520411A5 (https=) 2025-05-30
JPWO2022251208A5 true JPWO2022251208A5 (https=) 2025-05-30

Family

ID=84229086

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2023572721A Pending JP2024520411A (ja) 2021-05-24 2022-05-24 Ｘ連鎖性ミオチュブラーミオパチーの改善された治療のための組成物及び方法

Country Status (13)

Country Link
US (1) US20240366785A1 (https=)
EP (1) EP4351725A4 (https=)
JP (1) JP2024520411A (https=)
KR (1) KR20240025536A (https=)
AU (1) AU2022283262A1 (https=)
BR (1) BR112023024509A2 (https=)
CA (1) CA3219834A1 (https=)
CO (1) CO2023018156A2 (https=)
IL (1) IL308736A (https=)
MX (1) MX2023013926A (https=)
PH (1) PH12023553216A1 (https=)
TW (1) TW202306589A (https=)
WO (1) WO2022251208A1 (https=)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AR125077A1 (es) * 2021-03-11 2023-06-07 Audentes Therapeutics Inc Composiciones y métodos para tratar la miopatía miotubular ligada a x

Family Cites Families (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN104023727B (zh) * 2011-10-28 2017-04-05 鲁美纳医药公司 用于治疗小儿胆汁淤积性肝病的胆汁酸再循环抑制剂
US8957044B2 (en) * 2013-03-01 2015-02-17 Wake Forest University Health Sciences Systemic gene replacement therapy for treatment of X-linked myotubular myopathy (XLMTM)

Similar Documents

Publication Publication Date Title
Neri et al. The red half‐moon nail sign: a novel manifestation of coronavirus infection
Pozsgai et al. Systemic AAV-mediated β-sarcoglycan delivery targeting cardiac and skeletal muscle ameliorates histological and functional deficits in LGMD2E mice
US12357663B2 (en) Intrathecal delivery of recombinant adeno-associated virus encoding methyl-CpG binding protein 2
JP2023552443A (ja) ダノン病の治療
US20170252462A1 (en) Extended antegrade epicardial coronary infusion of adeno-associated viral vectors for gene therapy
JPWO2022251208A5 (https=)
US11952585B2 (en) Methods of treating phenylketonuria
Ishikawa et al. Cardiovascular considerations in the management of neuromuscular disease
RU2023134651A (ru) Композиции и способы улучшенного лечения х-сцепленной миотубулярной миопатии
JP2025523953A (ja) 肢帯型筋ジストロフィーのためのａｕｆ１遺伝子療法
Liu et al. Combined Insulin-Glibenclamide Therapy of NIDDM Patients in Primary Health Care: A follow-up study of its compliance and efficacy and a review of the literature
Williams et al. Some metabolic effects of thiouracil with particular consideration of adrenal functions
Russell, D.,* Fritz, V.,** Mieny, CJ,** Mendelsohn, D.,* Joffe, BI*** & Seftel Treatment of familial hypercholesterolaemia by partial ileal bypass
Scola et al. Amyloidotic muscle pseudohypertrophy: case report
JPWO2023150688A5 (https=)
RU2024125625A (ru) Композиции и способы для оптимизированного лечения болезни помпе
Khanna et al. P. 17.8 The co-formulation of pharmacological chaperone AT2220 with recombinant human acid alpha-glucosidase improves enzyme uptake and glycogen reduction in a mouse model of Pompe disease
Parsons Current Updates and Advances in Diagnosis and Treatment of Spinal Muscular Atrophy.
SCHEMAN et al. PSEUDOHYPERTROPHIC MUSCULAR DYSTROPHY: AN EVALUATION OF RECENT STUDIES
Dongsheng DWORF Gene Therapy for DMD
Lewis et al. AAV9 Gene Therapy in Type II GM1 Gangliosidosis—A Phase 1–2 Trial
Parsons Advances in the Management of Spinal Muscular Atrophy: Tailoring Treatment and Care Approaches to Improved Outcomes.
WO2026006391A1 (en) Compositions and methods for treating a heart disease
Unalp A Case of Juvenile Dermatomyositis Presenting with Respiratory Distress
Liberati et al. Clinical Gene Therapy Trials for Pompe Disease