According to The Times of Israel, a group of researchers believe they have found the origin of what could lead to a treatment for amyotrophic lateral sclerosis. Given the debilitating effect…
Several organisations are collaborating to develop a gene therapy that targets cystic fibrosis. Boehringer Ingelheim, Imperial Innovations, Oxford BioMedica, and the UK Cystic Fibrosis Gene Therapy Consortium will work…
The healthcare technology company RDMD was founded by Onno Faber, who has himself been diagnosed with a rare disorder. RDMD, which describes itself as “dedicated to accelerating research” with…
According to a story by The Japan Times, Masatane Muto relies on a power wheelchair to move around. He is hardly in control of his own body and requires help…
The Alagille Syndrome Alliance has launched its first ever awareness ribbon in support of the Alagille syndrome (ALGS) community! The multi-colored awareness ribbon is meant to represent the complex and uniquely…
According to a story from Charcot-Marie-Tooth News, a recent study has managed to identify new clinical evaluations that will allow doctors to more effectively monitor the condition of patients with…
According to a report by BioSpace, a new treatment for SCID could be around the corner. Mustang Bio, Inc. (Mustang), and St. Jude Children’s Research Hospital recently announced a partnership…
Sam Nicholson was a small child. His mother, Erica, did not find this surprising. Erica is only about five feet tall. She weighs about 100 pounds. She describes all of…
Erika Baker and her family have been dealing with the impact of adrenoleukodystrophy (ALD) for generations. The disease has impacted several of her family members, including her uncle, her cousins,…
According to a story reported by the New York Times, 5-year-old Garrett Matthias had already thought about what he wanted his funeral to be like. He didn't want it to…
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