SCD Features

sickle cell disease comorbidities

Features, SCD Features, Uncategorized

L-Glutamine in Sickle Cell Disease: The Real-World Impact to Date

By

Tori Rodriguez, MA, LPC, AHC

January 21, 2022

L-glutamine was approved in 2017 to treat acute complications in patients with sickle cell disease, but uptake of and adherence to it have been limited.

sickle cell disease

Features, SCD Features

An Often Overlooked Disease Exiting the Shadows

By

Kyle Habet, MD

January 10, 2022

Funding for sickle cell disease clinical trials and drug development has steadily increased and groundbreaking gene therapy research is being conducted.

Harsha K. Rajasimha

CC Features, Features, LAL-D Features, LCFAOD Features, SCD Features

Using Technology to Boost Ethnic, Geographic Diversity in Clinical Trials

By

Larry Luxner

October 27, 2021

Eliminating geographic and ethnic bias in clinical trials for rare diseases is the goal of Jeeva Informatics, says founder and CEO Harsha K. Rajasimha, PhD.

Features, LAL-D Features, MS Features, NMOSD Features, SCD Features

CORD Aims to Improve Diagnosis, Drug Access for Canada’s Rare Disease Patients

By

Larry Luxner

October 14, 2021

The Canadian Organization for Rare Disorders advocates for better diagnosis and drug access for Canada’s 3 million people with rare diseases.

Georgianne Arnold

Conferences, News Briefs, Pompe News Briefs, SCD Features, WODC 2021

Newborn Screening Saves Lives, Yet Raises Some Ethical Issues, Expert Says

By

Larry Luxner

August 27, 2021

Georgianne Arnold, MD, an expert on newborn screening, says the practice clearly saves lives, yet raises ethical concerns.

Next post in WODC 2021

Ensuring Access to Therapies for All Rare Disease Patients