Spinal deformities such as scoliosis and kyphosis are among the most serious complications of neurofibromatosis type 1 (NF1), a genetic disorder that affects about one in 3,000 people.

Medcare Women & Children Hospital has successfully administered a pioneering intrathecal gene therapy for Spinal Muscular Atrophy (SMA) to Hulus, a three-year-old patient from Turkey. This milestone ...

Even as OpenAI works to harden its Atlas AI browser against cyberattacks, the company admits that prompt injections, a type of attack that manipulates AI agents to follow malicious instructions often ...

Itvisma should only be administered intrathecally using a lumbar puncture by health care professionals experienced in performing the procedure. The Food and Drug Administration has approved Itvisma ® ...

Novartis has received approval from the U.S. Food and Drug Administration for Itvisma, a treatment for spinal muscular atrophy. Dr. John Day is professor of neurology and pediatrics, director of the ...

The FDA approved the intrathecal gene therapy onasemnogene abeparvovec (Itvisma) to treat spinal muscular atrophy (SMA) in adults and pediatric patients 2 years and older with a confirmed mutation in ...

The FDA has approved onasemnogene abeparvovec-brve (Itvisma, Novartis), an adeno-associated virus vector-based gene therapy for treatment of spinal muscular atrophy (SMA) in patients aged 2 years and ...

Novartis has received an FDA approval for Itvisma, a new version of the company’s gene therapy Zolgensma, to treat older patients with spinal muscular atrophy (SMA). The approval comes more than six ...

EAST HANOVER, N.J., Nov. 24, 2025 /PRNewswire/ -- Novartis today announced that the US Food and Drug Administration (FDA) has approved Itvisma® (onasemnogene abeparvovec-brve) for the treatment of ...

uniQure surged over 300% following strong 3-year data from its Huntington’s trial, yet remains undervalued due to skepticism over trial size and the use of external controls. AMT-130’s apparent ...