Clinical trial shows positive results for potential treatment to combat a rare immune-mediated disease
An international, Phase III clinical trial led by investigators at Mass General Brigham could improve the treatment of a rare ...
Medicover Hospitals Successfully Performs Marathwada's First Rare and Complex Simultaneous Liver-Kidney Transplant (SLKT)
Medicover Hospitals reaffirms its commitment to advanced and compassionate care. This case also underscores the profound impact of organ donation, a gift that can transform lives. He was diagnosed ...
European committee takes a second look at Alzheimer's drug and now says it should be approved
A European regulatory committee now recommends approval of the Alzheimer’s treatment lecanemab a few months after rejecting ...
We have the technology to save thousands of lives, experts say, and we must start using it now
Australia is on the brink of a healthcare revolution driven by DNA science that will save lives and prevent disease – but top ...
Cancer survivor claims she was dismissed for YEARS because doctors said she was 'too young' to get the disease
Katie Coleman, now 33, was diagnosed with a rare type of kidney cancer back in December 2020 after struggling to get doctors ...
FDA Grants Conditional Approval For PTC Therapeutics' Gene Therapy As First Ever With Direct Administration In Brain
PTC Therapeutics' Kebilidi gains FDA accelerated approval as the first brain-administered gene therapy in the U.S. for AADC ...
Arcturus: LUNAR-CF For CF Presses On With 1st Half Of 2025 Interim Data
Arcturus Therapeutics (ARCT) is continuing to evaluate the use of LUNAR-CF [ARCT-032] for the treatment of patients with Cystic Fibrosis [CF]. Read our latest report on the stock here.
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Fact Check: Did Miami Dolphins’ DT Really Donate Bone Marrow To Save His Sister? Everything You Need To Know
Jumping right into the concerning situation, Austin agreed to donate his bone marrow. Luckily, the bone marrow samples ...
Rare disease support: How to get help, resources, manage symptoms, find doctors
Are you battling a rare disease or think you might be suffering from one? If so, there are resources to get help from ...
Repurposed FDA-approved drug could uncover potential treatment for rare genetic disorders
In a new study, researchers at McMaster University have identified a potential treatment for Sandhoff and Tay-Sachs ...
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PTC wins FDA approval for first brain-delivered gene therapy Kebilidi
The FDA issued its first stamp of approval for a cell or gene therapy back in 2017 to Novartis' Kymriah. | The gene therapy ...
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Merck joins American Lung Association in raising awareness of pulmonary arterial hypertension
As the rollout of its recently launched pulmonary arterial hypertension (PAH) drug Winrevair ramps up, Merck is continuing ...