Intellectual disability-severe speech delay-mild dysmorphism syndrome

Disease Researchers

Specialists who have done research into Intellectual disability-severe speech delay-mild dysmorphism syndrome.

These specialists have recieved grants, written articles, run clinical trials, or taken part in organizations relating to Intellectual disability-severe speech delay-mild dysmorphism syndrome, and are considered knowledgeable about the disease as a result.

The people in this list are filtered based on their research related to Intellectual disability-severe speech delay-mild dysmorphism syndrome, and as a result may or may not have a clinical practice.

Name Location(s) Related Activity

Clinical Trials

A clinical trial is how pharmaceutical companies and the FDA determine if treatment for a rare disease is safe and effective. Because the number of patients with rare diseases are extremely small, it is difficult for the companies to enroll enough patients to statistically prove (that the improvement wasn't just by chance) that the treatment was effective. It may take many years to treat enough patients to determine if a treatment is effective. The FDA, patient communities, legislation, and the drug companies are working on ways to address this issue.




Title Status Principal Investigator First Posted Start Date

Source: ClinicalTrials.gov

NIH Grants

The National Institutes of Health sponsors a large number of grants each year in different areas of public health interest. As with all government spending, the funds allocated to each researcher, their research topic, and their results are available for review. InfoHub collects that data so you can see who is doing research in a particular area of rare diseases and what progress has been made.

Title Organization Principal Investigator(s) Fiscal Year
Source: NIH RePORTER

FDA Orphan Drugs

Orphan Drug is a special status given by the FDA to a medication that was specifically developed for treatment of a rare disease. This status provides pharmaceutical companies with financial incentives for developing and marketing the drug.

Patient assistance programs can help patients pay for expensive prescriptions. There are often eligibility restrictions based on income, insurance status, and citizenship.

Before taking any medication, always check with a qualified professional for healthcare information, treatment advice.




Generic Name Trade Name Use Status Date Designated Assistance Programs

Source: FDA Orphan Drug Database

FDA Orange Book Drugs

The FDA Orange Book drug database contains all approved drugs. Some drugs may be re-purposed from their original use to treat a rare disease. This table lists research papers that mention a drug in conjuntion with this disease.

Before taking any medication, always check with a qualified professional for healthcare information, treatment advice.

Drug Name Number of Articles

Source: FDA Approved Drug Products

Chemotext

Chemotext is a publicly-available Webserver that mines the published literature in PubMed in the form of Medical Subject Headings (MeSH) terms.

The goal of Chemotext is to enable text-based drug-target-disease relationships in order to identify novel drug repurposing candidates and discovery targets.