Latest news stories about centronuclear and myotubular myopathy can be found on this page. To see news stories from previous years please see the News archive pages.
- Tipp father’s joy as he raises funds needed for son’s emergency flight
Extra.ie, 28 December 2020 - Audentes: FDA Lifts Clinical Hold On Trial Of AT132 For Treatment Of X-Linked Myotubular Myopathy
Nasdaq, 27 December 2020 - FDA lifts clinical hold on Astellas AT132 trial
The Pharma Letter, 26 December 2020 - Audentes Therapeutics Announces FDA Lifts Hold on ASPIRO Clinical Trial of AT132 for Treatment of X-Linked Myotubular Myopathy (XLMTM)
Business Wire, 24 December 2020 - Sick baby’s return to New Zealand still to take flight
Stuff, 18 December 2020 - Impressive progress of little Jules
Nosy Media, 5 December 2020 - Tipperary man ‘humbled’ by support to help bring newborn with rare genetic disorder to New Zealand
Irish Examiner, 2 December 2020 - Couple in call for life-saving flight for baby Harrison
Extra.ie, 30 November 2020 - New Zealand baby with life-threatening illness stranded with parents in Ireland
NZ Herald, 27 November 2020 - Palmerston North family seeks help to bring home sick baby
Stuff, 20 November 2020 - Please help fly baby Harrison home
Tipperary Live, 19 November 2020 - Double celebration for Doonside resident
South Cost Sun, 18 November 2020 - Huge support for charity golf day in memory of baby Jack
Worthing Herald, 21 October 2020 - Living with CNM – a Doonside sufferer’s account
South Coast Sun, 13 October 2020
John Garrett Scoggin
The Daily News, 9 October 2020 - Dynacure to Present Disease Progression Model and Natural History Study Data at the 25th Annual Congress of the World Muscle Society (WMS25)
Yahoo Finance, 28 September 2020 - Dynacure to Present Disease Progression Model and Natural History Study Data at the 25th Annual Congress of the World Muscle Society (WMS25)
prnewswire.com, 28 September 2020 - Audentes’ rare disease gene therapy programme delayed after third patient death
pmlive.com, 25 August 2020 - Audentes Therapeutics Reports Third Death In A Clinical Trial
Technologytimes.pk, 22 August 2020 - Derailed gene therapy study reports 3rd death as safety and durability issues cloud a booming field
endpts.com, 21 August 2020 - Audentes Therapeutics Provides Update on the ASPIRO Clinical Trial Evaluating AT132 in Patients with X-linked Myotubular Myopathy
Pipelinereview.com, 21 August 2020 - Astellas’ Audentes reports 3rd death in gene therapy trial
Fiercebiotech.com, 21 August 2020 - Audentes Therapeutics provides update on the ASPIRO clinical trial evaluating AT132 in patients with X-linked myotubular myopathy
Business Wire, 21 August 2020 - Audentes Therapeutics Provides Update on the ASPIRO Clinical Trial Evaluating AT132 in Patients with X-linked Myotubular Myopathy
Audentes, 20 August 2020 - Dynacure receives rare pediatric disease designation from U.S. Food and Drug Administration for DYN101 for myotubular and centronuclear myopathies (CNM)
prnewswire.com, 30 July 2020 - Dynacure receives rare pediatric disease designation from U.S. Food and Drug Administration for DYN101 for myotubular and centronuclear myopathies
Dynacure, 30 July 2020 - Guard of honour and police motorbike escort for four year old boy after 14 months in hospital
ITV News, 24 July 2020 - Liverpool guard of honour after Hari’s long hospital stay
BBC News, 24 July 2020 - Caernarfon boy with myotubular myopathy leaves hospital
BBC News, 24 July 2020 - Hari Jones, who suffers from a rare muscular condition, returned home to Caernarfon from Alder Hey Children’s Hospital and was met with a police escort
North Wales Chronicle, 22 July 2020 - Tears outside hospital as little boy finally leaves after 14 months
Liverpool Echo, 22 July 2020 - On Being BOLD
LinkedIn, 10 July 2020 - Gene Therapy: It’s Time to Talk about High-Dose AAV
Genetic Engineering and Biotechnology News, 7 July 2020 - Concern following gene therapy adverse events
Medical Xpress, 2 July 2020 - Moving Forward after Two Deaths in a Gene Therapy Trial of Myotubular Myopathy
Genetic Engineering and Biotechnology News, 2 July 2020 - Audentes halts gene therapy trial after second patient death
The Pharma Letter, 30 June 2020 - Two boys’ deaths halt S.F. company’s rare disease gene therapy trial
Biz Journals, 29 June 2020 - Two patients die in Audentes gene therapy study, heightening concerns over high-dose treatments
Stat News, 28 June 2020 - Two patients die in Audentes gene therapy study, heightening concerns over high-dose treatments
Bio Pharma Dive, 26 June 2020 - Amid a Pandemic, Photographers Help Grieving Parents Find Hope Through Suffering
The Federalist, 6 April 2020 - Dynacure Adds $55M As RNA-Targeted Muscle Disorder Drug Enters Clinic
Xconomy, 3 April 2020 - Dynacure Announces €50M ($55M) Series C Financing
Biospace, 2 April 2020 - Dynacure Announces €50M ($55M) Series C Financing
Dynacure, 2 April 2020 - Dynacure Announces EUR50M ($55M) Series C Financing
P and T Community, 2 April 2020 - Funding supports ongoing Phase 1 / 2 ‘Unite-CNM’ study of DYN101 in Patients with Myotubular and Centronuclear Myopathies
Yahoo Finance, 2 April 2020 - An ’emergency’ birthday party for ten year old Cork boy
RTE News, 1 April 2020 - Dynacure Announces First Patient Dosed in Phase 1 / 2 ‘UNITE-CNM’ Study of DYN101 for the Treatment of Myotubular and Centronuclear Myopathies (CNM)
P and T Community, 5 March 2020 - Dynacure Announces First Patient Dosed in Phase 1 / 2 ‘UNITE-CNM’ Study of DYN101 for the Treatment of Myotubular and Centronuclear Myopathies (CNM)
BioSpace, 5 March 2020 - Dynacure Announces First Patient Dosed in Phase 1 / 2 ‘UNITE-CNM’ Study of DYN101 for the Treatment of Myotubular and Centronuclear Myopathies (CNM)
Dynacure, 5 March 2020 - Here’s how you can get involved in The Big Sunflower Project
Liverpool Echo, 4 March 2020 - Two Aurora Health Children’s patients head to meet the Brewers at Spring Training
TMJ4.com, 21 February 2020 - Family of twins call for change to DLA Motability scheme
Leigh Journal, 13 February 2020 - New insights from Mack Lab on mechanisms of gene therapy for x-linked myotubular myopathy
Institute for Stem Cell and Regenerative Medicine, 5 February 2020 - Chester-based Big Sunflower Project continues to grow in its 10th year
Chester Standard, 21 January 2020 - Astellas completes acquisition of Audentes Therapeutics
Audentes, 15 January 2020 - Astellas, Audentes merger may lead to new exon-skipping treatments for MD
Muscular Dystrophy News Today, 6 January 2020
