A graduate of the Harvard Business School, Raj Prabhakar is a dedicated business leader who has worked in the biopharmaceutical sector for over two decades. He serves as the Chief Business Officer and Senior Vice President at Rocket Pharmaceuticals, leading all business development, partnerships, and program management. One of Raj Prabhakar's various areas of interest is rare pediatric diseases.
Adrenoleukodystrophy (ALD) is a rare pediatric disease resulting from faulty genes. According to data from ChildrensHospital.org, one in 21,000 newborn males has an ALD mutation.
ALD is inherited through a faulty gene on the X chromosome. Children who suffer from ALD have high levels of long-chain fatty acids (VLCFAs) in their brains. These fatty acids damage the myelin sheath, a protective covering on some brain cells that facilitates the cell-to-cell transfer of signals. As a result, the brain loses its ability to send signals to some body parts.
Common symptoms of ALD include poor memory, behavioral problems, difficulty reading, an inability to understand speech, and aggression. Severe symptoms from disease progression include seizures, deafness, difficulty swallowing, poor coordination, and the inability to speak. Symptoms can appear as early as the age of four. ALD typically worsens between the ages of 4 and 8 and has historically resulted in permanent disability and death during this time period.
Males with ALD genes are susceptible to the detrimental manifestations of the mutation. Less than 50 percent of females born with the genetic mutation for ALD eventually develop symptoms and complications. Early diagnosis combined with a stem cell transplant can manage ALD.
Adrenoleukodystrophy (ALD) is a rare pediatric disease resulting from faulty genes. According to data from ChildrensHospital.org, one in 21,000 newborn males has an ALD mutation.
ALD is inherited through a faulty gene on the X chromosome. Children who suffer from ALD have high levels of long-chain fatty acids (VLCFAs) in their brains. These fatty acids damage the myelin sheath, a protective covering on some brain cells that facilitates the cell-to-cell transfer of signals. As a result, the brain loses its ability to send signals to some body parts.
Common symptoms of ALD include poor memory, behavioral problems, difficulty reading, an inability to understand speech, and aggression. Severe symptoms from disease progression include seizures, deafness, difficulty swallowing, poor coordination, and the inability to speak. Symptoms can appear as early as the age of four. ALD typically worsens between the ages of 4 and 8 and has historically resulted in permanent disability and death during this time period.
Males with ALD genes are susceptible to the detrimental manifestations of the mutation. Less than 50 percent of females born with the genetic mutation for ALD eventually develop symptoms and complications. Early diagnosis combined with a stem cell transplant can manage ALD.
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