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News > Research
The FDA Just Gave a Rare Pediatric Gene Therapy Its Fastest Regulatory Track. Here's What Families of Children with NGLY1 Deficiency Need to Know.
The U.S. Food and Drug Administration granted Regenerative Medicine Advanced...multiple body systems and is characterized by global developmental delay, cognitive impairment ranging from mild...a regulatory pathway designed to expedite the development and review of regenerative medicine therapies...
Research > Drug Development
Should Your Child Participate in a Clinical Trial? A Parent's Decision Framework
...don't fully understand, for a drug that may or may not work, with...side effects, and whether the drug is tolerated. This is the highest-risk...the highest-risk phase. The drug may not work at all; safety...looking for a signal that the drug does what it's supposed to do....3,000 participants or more. The drug has shown promise in earlier...
Research > Drug Development
Fragile X Drug Trials: The First Real Breakthroughs and What They Mean for Families
If you've been following Fragile X drug research for any length of time, you've probably learned to hold good news...it carefully rather than cautiously. In 2024, Zatolmilast became the first drug to produce unequivocally positive results in a Phase 2 trial for Fragile...
Research > Drug Development
The Drug Development Pipeline for Special Needs: What Parents Need to Know About How Treatments Go from Lab to FDA Approval
...about a promising autism drug entering Phase 2 trials,...Understanding where a drug sits in the development...where a drug sits in the development pipeline matters when...question about whether a drug is safe enough and effective...Testing Begins. Before a drug is tested in people, it...an Investigational New Drug (IND) application, which...
Research > Funding
Investing in the Future of Special Needs Research and Innovation
...Technologies. One key area where research funding makes a big impact is in the development of cutting-edge assistive technologies. These devices and software help...Breakthroughs in genetics, neuroscience, and other fields have led to targeted drug therapies and interventions for conditions like fragile X syndrome, Down...
Research > Drug Development
Unveiling Breakthroughs in Muscular Dystrophy Treatment
...New drugs Are Developed. Making a new drug is a long process. It can take 10-15...Next, they make and test different drug compounds. The most promising ones move...ones move on to animal testing. If a drug seems safe and effective, it can be...check the safety and benefits of the drug. The FDA looks at all the data before...
Parenting > Adulthood
Adopting a Child with Special Needs
...medically fragile children with complicated syndromes to those at risk for developmental, behavioral, or learning issues due to pre-natal exposure to drugs or...medical issues, while a teacher feels prepared to parent a child who was drug-exposed and at risk for learning and behavior...
Health > Treatment
Is There a New Drug for Cerebral Palsy?
...suggests the use of a new drug that might limit the development...drug that might limit the development of cerebral palsy if administered...palsy were administered a drug that targets and treats...who did not receive the drug. If the new drug works...the drug. If the new drug works for humans as well...effects. Researchers say the drug must be administered right...
Research > Drug Development
Turning Basic Research into Better Treatments
...myelin repair for MS, a new and comprehensive model for medical research and drug development that significantly shortens the time to market for new medicines...repair for MS, a new and comprehensive model for medical research and drug development that significantly shortens the time to market for new medicines for all...
Research > Drug Development
Muscular Dystrophy Drug Success
Ohio State Univ. researchers find drugs lisinopril and spironolactone help preserve muscle in DMD, potentially extending lives.
Health > Mental Health
New Mental Health Manual Could Be “Dangerous”
...signed a petition asking that the fifth edition of the DSM be halted in its development and rethought. The new DSM could also give serial rapists and sex abusers...difficult to avoid the conclusion that DSM-5 will help the interests of the drug companies. “Madness and misery exist, but they come in many shapes and...
Special Needs > Attention Deficit Disorders
ADD/HD and US Healthcare: Fabulous or Just Funded?
...when it comes to our research and development (R&D) in the area of drug creation....and development (R&D) in the area of drug creation. Funding directs the R&D....creation. Funding directs the R&D. Drug companies have a great influence on...presented to us?" You may ask. Well, since drug companies lobbyists spent the most...