Archive for clinical trials
Risdiplam improves motor function in infants with spinal muscular atrophy
Until recently, babies and children with spinal muscular atrophy (SMA) lacked any type of treatment capable of stopping the neuromuscular disease from progressing. In SMA, damaged motor neurons in the spinal cord fail to correctly send messages to the muscle cells. As a result, infants and children with SMA may not be able to hold ... Read More about Risdiplam improves motor function in infants with spinal muscular atrophy
ADHD drug class leads to fewer side effects in preschool children
About 2.4 percent of preschool-age children have an attention-deficit/hyperactivity disorder (ADHD) diagnosis. For these children, behavioral interventions intended to redirect a child or otherwise replace negative behaviors with positive ones are the first line of treatment. But what if symptoms linger, or are so severe that they interfere with a child’s social, emotional, and educational ... Read More about ADHD drug class leads to fewer side effects in preschool children
COVID-19 vaccines for kids: What has to happen first
Now that adults and older teens are receiving COVID-19 vaccines, some younger kids are wondering why they can’t get vaccinated too. While testing has begun in children 6 months to age 12, we aren’t ready to begin vaccinating most kids yet. But we do know the steps the companies are taking to be sure their ... Read More about COVID-19 vaccines for kids: What has to happen first
What’s the difference between the COVID-19 vaccines?
After a long, difficult year, the world had reason to celebrate in late 2020 when the U.S. Food and Drug Administration (FDA) authorized two COVID-19 vaccines for emergency use. Many more COVID-19 vaccines are in various stages of development. Several of these are in the final stage of clinical trials and could be submitted for ... Read More about What’s the difference between the COVID-19 vaccines?
It’s personal: How the Boston Children’s progeria research community brought new life to an old drug
In late November, the U.S. Food and Drug Administration (FDA) approved a repurposed cancer drug called lonafarnib to treat Hutchinson-Gilford progeria syndrome, better known as progeria — an ultra-rare incurable genetic disease associated with rapid aging. On average, children with progeria die before age 15, usually the result of rapidly accelerated atherosclerosis resulting in heart ... Read More about It’s personal: How the Boston Children’s progeria research community brought new life to an old drug
Poverty predicts worse cancer outcomes, even in children receiving top-tier care
A pair of recent studies suggests that even among patients receiving advanced cancer care, poverty is a predictor of worse outcomes. The disturbing findings raise many questions that follow-up studies are now exploring. Key takeaways Poverty was associated with 3.7-fold higher mortality among children receiving targeted immunotherapy for high-risk neuroblastoma. Household poverty was associated with ... Read More about Poverty predicts worse cancer outcomes, even in children receiving top-tier care
The new COVID-19 vaccines: Will they be safe?
Just within the past week, pharmaceutical companies have announced encouraging news about two COVID-19 vaccines in the final development phase. As more vaccine developers announce similar reports in the coming weeks and months, it’s key to remember that vaccine safety is as important as effectiveness against the virus. Fortunately, scientists have learned lessons from the ... Read More about The new COVID-19 vaccines: Will they be safe?
Remdesivir: What to know about the first drug approved to treat COVID-19
In late October, the U.S. Food and Drug Administration (FDA) officially approved the antiviral drug remdesivir (Veklury) for COVID-19. The approval has been controversial, with many scientists questioning the strength of the evidence and proposing that the drug undergo further clinical trials before receiving full FDA approval . What does this mean for children who ... Read More about Remdesivir: What to know about the first drug approved to treat COVID-19
A diabetes drug with a potential side benefit: Reduced COVID-19 mortality
In 2006, the Food and Drug Administration approved the drug sitagliptin to lower blood sugar in type 2 diabetes. Now, a multicenter observational study in Italy suggests the drug also cuts mortality in diabetic patients hospitalized with COVID-19 — by as much as half. Paolo Fiorina, MD, PhD, of Boston Children’s Hospital led the study, ... Read More about A diabetes drug with a potential side benefit: Reduced COVID-19 mortality
Global partnership leads to improved treatment for childhood diarrhea
Diarrheal diseases remain a major childhood health problem in many parts of the world. Each year, they lead to the deaths of more than 500,000 children under age 5 globally. A new large clinical trial based in Tanzania and India, led by investigators at Boston Children’s Hospital, local research universities, and the World Health Organization ... Read More about Global partnership leads to improved treatment for childhood diarrhea