MDA Kickstart Program Receives FDA Rare Pediatric Disease Designation and Orphan Drug Designation for Congenital Myasthenic Syndrome
The Muscular Dystrophy Association (MDA) proudly announces that its groundbreaking MDA Kickstart program has received both an FDA Rare Pediatric Disease designation and Orphan Drug Designation for ...
Researchers identify possible treatment for rare disorder CTNNB1 syndrome
Researchers at Tufts University School of Medicine and the Graduate School of Biomedical Sciences (GSBS) have identified a ...
Biogen Still Has Competitive Advantages in Neurology, but Its Moat Looks Narrow
We think Biogen's neurology, immunology, and focus on rare diseases support a narrow moat. Biogen's strategy has its roots in the 2003 merger of Biogen (Avonex for treating multiple sclerosis, or MS) ...
STAT3d
In a bid to treat their children’s rare diseases, these three parents founded companies
At #STATSummit, three parents discussed the challenges they've faced trying to find treatments for their children with rare ...
My Doctors Think I Have Stiff-Person Syndrome. These Were the Symptoms I Missed
Carrie Jean Robinette, 46, of San Diego, has always had a lot of health issues, but she didn’t think she had a serious ...
The Hill14d
Rare comet visible in the night sky won’t return for 80,000 years
Comet Tsuchinshan-ATLAS, also known as C/2023 A3 to scientists and pronounced Choo-cheen-shahn, is expected to be visible to the naked eye across the Northern Hemisphere for several weeks in ...
Morningstar14d
NMD Pharma to Present Data on its Skeletal Muscle Targeted Therapy for Rare Neuromuscular Diseases at the 2024 AANEM Annual Meeting
NMD Pharma to Present Data on its Skeletal Muscle Targeted Therapy for Rare Neuromuscular Diseases at the 2024 AANEM Annual Meeting Aarhus, Denmark, 11 October 2024 – NMD Pharma A/S, a clinical ...
Everyday Health14d
Beyond Pain: How to Cope With Rare Fibromyalgia Symptoms
Rare fibromyalgia symptoms include sensitivity to touch, paresthesia, and more. Learn expert-backed coping strategies.
Mena FN14d
NMD Pharma To Present Data On Its Skeletal Muscle Targeted Therapy For Rare Neuromuscular Diseases At The 2024 AANEM Annual Meeting
(MENAFN- GlobeNewsWire - Nasdaq) NMD Pharma to Present Data on its Skeletal Muscle Targeted Therapy for Rare Neuromuscular Diseases at the 2024 AANEM Annual Meeting Aarhus, Denmark, 11 October ...
manilatimes14d
NMD Pharma to Present Data on its Skeletal Muscle Targeted Therapy for Rare Neuromuscular Diseases at the 2024 AANEM Annual Meeting
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