Fragile X syndrome

Zynerba flunks another PhII trial, all but ending Fragile X hopes
Zynerba flunks another PhII trial, all but ending Fragile X hopes
Endpoints
Zynerba
CBD
Fragile X syndrome
clinical trials
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Ovid's lead drug shows promising efficacy signal in Fragile X syndrome study, but optimal dosing regimen is unclear for path forward
Ovid's lead drug shows promising efficacy signal in Fragile X syndrome study, but optimal dosing regimen is unclear for path forward
Endpoints
Ovid Therapeutics
Takeda
Lundbeck
Fragile X syndrome
clinical trials
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Design Therapeutics launches with $45m to develop a new class of disease-modifying therapies for serious degenerative disorders
Design Therapeutics launches with $45m to develop a new class of disease-modifying therapies for serious degenerative disorders
Pharmaceutical Business Review
Design Therapeutics
degenerative diseases
Fragile X syndrome
myotonic muscular dystrophy
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Months after $10M Series A, rare disease AI upstart Healx hauls in $56M in fresh funding
Months after $10M Series A, rare disease AI upstart Healx hauls in $56M in fresh funding
Endpoints
Healx
Fragile X syndrome
autism
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Tetra Discovery Partners Initiates Ph II Trial of Fragile X Syndrome Drug
Tetra Discovery Partners Initiates Ph II Trial of Fragile X Syndrome Drug
CP Wire
Tetra Discovery Partners
Fragile X syndrome
BPN14770
clinical trials
orphan drugs
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Tetra Discovery Partners Initiates Ph II Trial of Fragile X Syndrome Drug
Tetra Discovery Partners
Fragile X syndrome
BPN14770
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Diabetes drug metformin shows promise in treating one form of autism
Diabetes drug metformin shows promise in treating one form of autism
Fierce Biotech
diabetes
metformin
autism
Fragile X syndrome
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Marinus Pharmaceuticals gets a boost from the FDA
Marinus Pharmaceuticals gets a boost from the FDA
Bizjournals.com
Marinus Pharma
FDA
Fragile X syndrome
ganaxolone
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Alcobra's genetic disorder drug fails in mid-stage trial
Alcobra's genetic disorder drug fails in mid-stage trial
Yahoo/Reuters
Alcobra
Fragile X syndrome
autism
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Roche abandons another Fragile X R&D program after PhII trials flunk out
Roche abandons another Fragile X R&D program after PhII trials flunk out
Fierce Biotech
Roche
Fragile X syndrome
R&D
RG7090
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Alcobra Starts Patient Enrollment for Mid-Stage Study on MDX
Alcobra Starts Patient Enrollment for Mid-Stage Study on MDX
Yahoo/Zacks.com
Alcobra
MDX.V
Fragile X syndrome
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