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The Latest Breaking News from Gene Therapy

Sarepta Catapults 30% After Hitting A 'Grand Slam' Approval In Muscular Dystrophy

Sarepta stock rocketed Friday after hitting a "grand slam" approval that expands the market for its Duchenne muscular dystrophy gene therapy more than tenfold, according to one estimate.

Investors Business Daily

Sarepta Jumps After Pfizer's Rival Gene Therapy Flops In Phase 3 Test

Sarepta stock jumped Thursday after Pfizer's rival gene therapy for Duchenne muscular dystrophy failed in final-phase testing.

Investors Business Daily

When there’s no commercial incentive to develop gene therapy – hospitals will try to fill the gap

Individually, rare diseases are rare, but there are so many rare diseases that over 400 million people are affected worldwide. Together, rare diseases aren’t so rare.

The Conversation

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Children born deaf can hear in both ears in world-first gene therapy trial

Given as an infusion into the ear, the treatment works by replacing the faulty DNA which causes a type of inherited deafness known as DFNB9.

The Independent UK

Gene therapy trial gives deaf children hearing in both ears

Study participants born unable to hear could locate sound sources, recognise speech and dance to music after treatment

The Guardian - UK

Two deaf children can hear in both ears after world-first treatment

After the gene therapy, the children went from being profoundly deaf to having mild to moderate hearing loss

The Independent UK

Argentinian couple moves to US to allow their toddler to join gene therapy trial

The Lovatos relocated from Guatemala to Ohio for their son, who has the rare genetic disorder Canavan disease

The Guardian - US

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Deaf baby can hear after 'mind-blowing' gene therapy treatment

Seven months after her treatment, the baby girl can now respond to her parents' voices without the aid of a cochlear implant.

LiveScience

Great Ormond Street hoping to license gene therapy for ‘bubble baby’ syndrome

Hospital to take unprecedented step after drug firm pulled out despite successful trial of treatment

The Guardian - UK

UK toddler has hearing restored in world first gene therapy trial

Opal Sandy can hear almost perfectly after groundbreaking surgery that took just 16 minutes

The Guardian - UK

Pfizer reports patient death in Duchenne gene therapy study

Pfizer's gene therapy trial for Duchenne muscular dystrophy resulted in a young patient's death from cardiac arrest

The Hindu

Pfizer Wins FDA Approval For $3.5 Million Gene Therapy, Rivaling CSL, Uniqure

The FDA approved Pfizer's hemophilia gene therapy on Friday. But Pfizer stock only inched ahead on the approval early Friday.

Investors Business Daily

3 Standout Penny Stocks to Watch This April

Investing in penny stocks carries substantial risk, but also offers the potential for significant returns. These penny stocks, all with bullish analyst ratings, have considerable upside potential, according to Wall Street.

Barchart

Gene Therapy Breakthroughs In The 1990S Revolutionized Medical Treatment.

Explore the pivotal era of gene therapy in the 1990s, from groundbreaking advancements to significant challenges, shaping modern regenerative medicine.

Top News

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Investors Business Daily

How Sleeper Biotech Solid Biosciences, With A 687% Gain, Is Making A Comeback

Solid Biosciences is shrugging off several messy years in gene therapy development. The biotech stock has gotten a triple-digit boost.

Top News

Kyowa Kirin's Gene Therapy: Most Expensive US Drug

Kyowa Kirin's .3 million gene therapy sparks debates on accessibility and affordability in healthcare.

Top News

Kyowa Kirin Gene Therapy: Most Expensive US Drug At .3M

Kyowa Kirin's .3 million gene therapy sparks debate on accessibility and pricing of innovative treatments in the US.

Top News

Ensuring Quality Control In Cell And Gene Therapy Manufacturing

Learn about the importance of quality control in cell and gene therapy manufacturing for patient safety and market success.

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The Hindu

German firm Miltenyi Biotec opens office, plans Cell and Gene Therapy CoE

Miltenyi Biotec launches operations in Hyderabad, investing in a Cell and Gene Therapy centre of excellence.

Top News

Exploring The Top 10 Genetic Engineering Breakthroughs

Explore the latest breakthroughs in genetic engineering, from CRISPR-Cas9 to gene therapy, and their impact on healthcare and conservation.

Dynamite News

Heart Attack: Gene therapy improves advanced heart failure in animal model

During a heart attack, blood stops flowing into the heart. Without oxygen, a portion of the heart muscle dies. Read further on Dynamite News:

Dynamite News

Gene therapy may slow progression of a life-threatening cardiac ailment

The investigational treatment targets the loss of function of a gene implicated in many cases of ARVC, plakophilin-2 (PKP2). Read further on Dynamite News:

The Guardian - UK

Gene therapy hailed as ‘medical magic wand’ for hereditary swelling disorder

Single-dose treatment transformed lives of patients with potentially deadly condition in first human trial

LiveScience

Gene therapies restore hearing in several kids with inherited deafness

Two new gene therapies show promise in treating otoferlin-related deafness, an inherited condition.

WEKU

Gene therapy shows promise for an inherited form of deafness

Scientists report that gene therapy restored at least some hearing and speech for five out of six children with a rare form of genetic deafness.

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