HealthQuill GEne therapy
Ultrasound of a baby
Drugs Health

Fetus treated with risdiplam for rare genetic disorder, a medical first

HQ Team February 27, 2025: A study published in The New England Journal of Medicine (NEJM) reveals that administration of the oral drug.

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Regeneron Pharmaceuticals, Inc.’s investigational gene therapy improved hearing in 10 out of 11 children born with hearing loss because of gene mutation, according to a company statement.
Drugs Health Medical

Regeneron’s trial therapy improves hearing in children with gene mutations

Regeneron Pharmaceuticals, Inc.’s investigational gene therapy improved hearing in 10 out of 11 children born with hearing loss because of gene mutation, according.

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Moderna Inc., a biotechnology company, and Merck announced that an experimental personalised cancer vaccine, based on the messenger RNA (mRNA) technology, has received Breakthrough Therapy Designation from the USFDA.
Health Research

Breakthrough system allows mRNA delivery through blood-brain barrier

February 18, 2025: Scientists at the Icahn School of Medicine at Mount Sinai have achieved a major breakthrough in brain therapy by developing.

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China’s YolTech Therapeutics, a biotechnology startup, has started its clinical trial of an investigational therapy for treating transfusion-dependent beta-thalassemia (TDT), a severe genetic blood disorder.
Drugs Health Medical

China’s YolTech starts gene editing trial for treating a blood disorder 

China’s YolTech Therapeutics, a biotechnology startup, has started its clinical trial of an investigational therapy for treating transfusion-dependent beta-thalassemia (TDT), a severe genetic.

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Pfizer Inc., got a nod from the US drug regulator for its drug Hympavzi to treat two types of a rare blood disorder in haemophilia patients.
Drugs Health Pharma

Pfizer’s drug to treat rare haemophilia disorder gets US regulator’s approval

Pfizer Inc., got a nod from the US drug regulator for its drug Hympavzi to treat two types of a rare blood disorder.

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Drugs Health Medical

Breakthrough gene therapy restores hearing in children with inherited deafness

HQ Team August15, 2024: A clinical trial has successfully restored hearing in five children suffering from inherited deafness by administering gene therapy to.

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An 18-month-old baby girl, Opal Sandy, who was born deaf got back her hearing ability after gene therapy — the youngest child to receive such treatment.
Drugs Health Pharma

Deaf 18-month girl gets back hearing ability after gene therapy

HQ Team May 15, 2024: An 18-month-old baby girl, Opal Sandy, who was born deaf got back her hearing ability after gene therapy.

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Drugs Health Medical

Gene therapy restores hearing in children with inherited deafness

Bharti Jayshankar January 25, 2024: A new gene therapy has demonstrated remarkable success in treating inherited deafness. Two separate studies, one conducted by.

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Voyager Therapeutics, Inc., will receive an upfront payment of $100 million from Switzerland-based Novartis AG to develop gene therapies for two genetic disorders.
Drugs Health Pharma

Novartis to pay $100m upfront to Voyager for gene therapies

HQ Team January 3, 2023: Voyager Therapeutics, Inc., will receive an upfront payment of $100 million from Switzerland-based Novartis AG to develop gene.

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The receptor of Oxytocin, a hormone considered essential to forming social bonds, may not play the role that scientists have assigned it for the past 30 years, according to US scientists.
Drugs Health Medical

Opinion: Sickle Cell Disease and gene therapy

By K. Leelamoni December 19, 2023: Recent reports about the FDA’s approval of gene therapy to treat Sickle Cell Anemia mark a major.

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