Clinical Resources

PubMed is a service of the U.S. National Library of Medicine that includes citations and abstracts (if available) from MEDLINE and other life science journals back to 1948.

PubMed Central
Pub Med Central is the U.S. National Library of Medicine’s digital archive of biomedical and life sciences journal literature, providing free access to the full text of articles.

Clinical Trial Toolbox

Code of Federal Regulations (CFR)
The CFRs are the rules for the United States organized by Title. Title 21 is specific to Food and Drug law and is split into parts ranging from electronic records (Part 11), human subject protection (Part 50), investigational new drug applications(Part 312) to orphan drugs (Part 316). Search CFR Title 21.

The International Conference on Harmonization of Technical Requirements for Registration of Pharmaceuticals for Human Use (ICH)
The ICH is made up of representatives from regulatory agencies in the United States, Japan and Europe and representatives from the pharmaceutical industry. The ICH has developed multiple guidelines that recommend a streamlined process in the drug development, testing and marketing process. ICH guidelines.

Investigational New Drug (IND) Application.
An IND application is required for the study of any non marketed drug and for any marketed drug used off label in a clinical trial.
This site provides information on federal and privately funded clinical trials.

Common Terminology Criteria for Adverse Events
The CTCAE is an adverse event (AE) coding system developed for oncology trials by the National Cancer Institute. The MSG uses this coding system in some of its trials to standardize AE coding.

Related Consortia

CINCH (Clinical Investigation of Neurologic Channelopathies Consortium) CINCH is a collaborative effort of doctors, patient organizations, and federal health agencies that want to learn more about these diseases so that ultimately they can offer better treatments to patients.

NEALS (The Northeast Amyotrophic Lateral Sclerosis Consortium)

PNCR (Pediatric Neuromuscular Clinical Research Network)
The PNCR Network brings together outstanding clinical investigators at four leading academic institutions in the Northeast and provides an opportunity for patients with SMA in this region of the United States to actively participate as research participants in a search for the cure of this devastating childhood motor neuron disease.

The Fields Center
The Fields Center seeks to provide individuals with FSHD and their families with useful information about FSHD.

TREAT-NMD is a network for people with neuromuscular diseases and professionals working in the field.

BIO-NMD is an EU funded project which bridges basic research and clinical research with the aim of discovering and validating biomarkers for neuromuscular disease.

Funding sources

Muscular Dystrophy Association
MDA is the nonprofit health agency dedicated to curing muscular dystrophy, ALS and related diseases by funding worldwide research. The Association also provides comprehensive health care and support services, advocacy and education.

Food and Drug Administration
The FDA Office of Orphan Products Development’s (OOPD) mission is to assist and encourage the identification, development, and availability of safe and effective products for people with rare diseases/disorders. The OOPD administers the major provisions of the Orphan Drug Act (ODA) which provide incentives for sponsors to develop products for rare diseases, usually defined as affecting fewer than 200,000 people in the United States.

National Institutes of Health
NIH is the steward of medical and behavioral research for the Nation. Its mission is science in pursuit of fundamental knowledge about the nature and behavior of living systems and the application of that knowledge to extend healthy life and reduce the burdens of illness and disability.

  • NINDS – The mission of NINDS is to reduce the burden of neurological disease – a burden borne by every age group, by every segment of society, by people all over the world.
  • NIAMS – The mission of the National Institute of Arthritis and Musculoskeletal and Skin Diseases is to support research into the causes, treatment, and prevention of arthritis and musculoskeletal and skin diseases, the training of basic and clinical scientists to carry out this research, and the dissemination of information on research progress in these diseases.

Congressionally Directed Medical Research Programs

The Congressionally Directed Medical Research Programs (CDMRP) was initiated by a Congressional appropriation to foster novel approaches to biomedical research in response to the expressed needs of its stakeholders – the American public, the military, and Congress. The CDMRP fills research gaps by funding high impact, high risk and high gain projects that other agencies may not venture to fund. While individual programs are unique in their focus, all of the programs managed by the CDMRP share the common goal of advancing paradigm shifting research, solutions that will lead to cures or improvements in patient care, or breakthrough technologies and resources for clinical benefit. The CDMRP strives to transform healthcare for Service Members and the American public through innovative and impactful research.

ALSRP – The mission for the ALSRP is to fund innovative and impactful research to develop new treatments for ALS.

DMDRP – The mission for the DMDRP is to better support discovery and development of therapeutics, devices, and other interventions, and to promote their rigorous clinical testing.

PRMRP – The mission for the PRMRP is to encourage, identify, select, and manage medical research projects of clear scientific merit and direct relevance to military health. The PRMRP is committed to supporting research that has the potential to profoundly impact the development and implementation of medical devices, drugs, and clinical guidance that will enhance the precision and efficacy of prevention, diagnosis, and treatment across a wide range of disciplines including autoimmune diseases and immunology, cardiovascular health, endocrine health and metabolism, environmental health, infectious diseases, internal medicine, neurological and psychological health, orthopedic and regenerative medicine, and respiratory health and injury.