Duchenne Muscular Dystrophy Exon Deletion

Duchenne, Solid Biosciences and Positive Trial Data

Duchenne gene therapy from Solid Biosciences produces promising results in early study

Solid said that the first patients to receive its Duchenne gene therapy all produced high levels of the target protein, an early sign of promise

Seeking Alpha on MSN · 10h

Solid Bio surges after trial data for lead asset in Duchenne

Solid Biosciences (SLDB) stock surges after strong Phase 1/2 data for its Duchenne therapy SGT-003. Read more here.

MarketWatch · 9h

Solid Biosciences Shares Rise After Positive Trial Data for SGT-003 in Duchenne MD

Solid Biosciences shares were up 50% to $6.06 after the company said it saw positive initial data from a Phase 1/2 trial evaluating SGT-003, a gene therapy product candidate intended for the treatment of Duchenne muscular dystrophy.

pharmaphorum3mon

Sarepta pulls plug on Duchenne exon-skipping drug

a follow-up to its Duchenne muscular dystrophy (DMD) therapy Exondys 51. The drug – formerly known as SRP-5051 – is a more potent version of Exondys 51 (eteplirsen), an exon-skipping therapy ...

STAT12d

Remembering a remarkable Duchenne champion

Patient advocate Austin Leclaire, who died Feb. 1, played a pivotal role in spurring the development of the first medicines ...

Managed Healthcare Executive6d

Sarepta Announces Positive Results for Elevidys, Gene Therapy for Duchenne

Results show improved functional outcomes for people with Duchenne muscular dystrophy who are ambulatory. Elevidys, which is ...

pharmaphorum3d

Nippon Shinyaku’s DMD drug fails confirmatory trial

The future of Nippon Shinyaku’s Duchenne muscular dystrophy (DMD ... for DMD patients to stand up from a supine position. The exon-skipping drug was approved by the FDA for DMD patients with ...

Nasdaq15d

Entrada Therapeutics Authorizes Phase 1/2 Clinical Trial for ENTR-601-44 to Treat Duchenne Muscular Dystrophy, On Track for Q2 2025 Initiation

clinical study of ENTR-601-44 for the potential treatment of Duchenne muscular dystrophy (DMD) in patients with a confirmed mutation in the gene amenable to exon 44 skipping. “The clearance by ...

Investor's Business Daily on MSN4mon

Avidity Biosciences, Up 441%, Nears Another Record High As Dystrophy Space Heats Up

Avidity Biosciences is also testing treatments for myotonic dystrophy type 1 and a subset of patients with Duchenne ...

The Hindu1y

Indo-Japanese researchers develop ‘disease-modifying’ treatment for Duchenne’s Muscular Dystrophy

A team of doctors from Tamil Nadu along with scientists from Japan have developed a disease-modifying treatment for Duchenne Muscular Dystrophy ... were gene therapy, Exon-skipping and disease ...

15don MSN

Entrada Therapeutics advances Duchenne treatment study

BOSTON - Entrada Therapeutics , Inc. (NASDAQ:TRDA) received authorization from the UK's Medicines and Healthcare Products Regulatory Agency (MHRA) to initiate a Phase 1/2 clinical trial for its ...

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